bims-mitdis Biomed News
on Mitochondrial disorders
Issue of 2026–02–22
sixty-nine papers selected by
Catalina Vasilescu, Helmholz Munich



  1. Nat Metab. 2026 Feb 16.
      Dihydroxyacetone phosphate (DHAP), glycerol-3-phosphate (Gro3P) and reduced/oxidized nicotinamide adenine dinucleotide (NADH/NAD⁺) are key metabolites of the Gro3P shuttle, which transfers reducing equivalents between the cytosol and mitochondria. Targeted activation of Gro3P biosynthesis has recently emerged as a promising strategy to alleviate reductive stress. However, because Gro3P constitutes the backbone of triglycerides, its accumulation can promote extensive lipogenesis. Here we show that a genetically encoded tool based on a di-domain glycerol-3-phosphate dehydrogenase from the alga Chlamydomonas reinhardtii (CrGPDH) effectively operates both the alternative Gro3P shunt, which regenerates NAD⁺ while converting DHAP to Gro3P, and the glycerol shunt, which converts Gro3P to glycerol and inorganic phosphate, across transformed and primary mammalian cell cultures as well as mouse liver. CrGPDH expression supported proliferation of cancer cells under respiratory chain inhibition or hypoxia, as well as patient-derived fibroblasts with mitochondrial dysfunction. Moreover, CrGPDH decreased triglyceride levels in kidney cancer cell lines and reversed ethanol-induced triglyceride accumulation in mouse liver. Thus, CrGPDH represents a promising xenotopic tool to alleviate redox imbalance and associated impaired lipogenesis in conditions ranging from primary mitochondrial diseases to steatosis.
    DOI:  https://doi.org/10.1038/s42255-025-01450-3
  2. Nat Metab. 2026 Feb 17.
      Dihydroorotate dehydrogenase is a rate-limiting enzyme of de novo pyrimidine synthesis. In most eukaryotes, this enzyme is bound to the inner mitochondrial membrane, where it couples orotate synthesis to ubiquinone reduction. As ubiquinone must be regenerated by respiratory complex III, pyrimidine biosynthesis and cellular respiration are tightly coupled. Consequently, inhibition of respiration suppresses DNA synthesis and cell proliferation. Here we show that expression of the Saccharomyces cerevisiae URA1 gene (ScURA) in mammalian cells uncouples pyrimidine biosynthesis from mitochondrial electron transport. ScURA forms a homodimer in the cytosol that uses fumarate as an electron acceptor instead of ubiquinone, enabling respiration-independent pyrimidine biosynthesis. Cells expressing ScURA are resistant to drugs that inhibit complex III and the mitochondrial ribosome. Additionally, ScURA enables growth of mitochondrial-DNA-lacking ρ0 cells in uridine-deficient medium and ameliorates the phenotype of cellular models of mitochondrial diseases. Overall, this genetic tool uncovers the contribution of pyrimidine biosynthesis to the phenotypes arising from electron transport chain defects.
    DOI:  https://doi.org/10.1038/s42255-026-01454-7
  3. J Family Community Med. 2026 Jan-Mar;33(1):33(1): 47-52
      Mitochondrial diseases are considered one of the most common groups of neurogenetic diseases. Complex I (CI) deficiency is the most encountered single enzyme deficiency of the mitochondrial diseases. The mutation of the NDUFA is linked to Leigh syndrome and CI defects. This article reports on a patient with mutation in NDUFA12 that was initially perceived as idiopathic intracranial hypertension, calling attention to the importance of considering NDUFA12 mutations in optic atrophy and dystonia diagnoses, particularly in young patients with new onset headache and progressive bilateral visual impairment. In addition, it emphasizes the need to explore other nonneurological features in diagnosing mitochondrial disease in those who do not fit into a defined syndrome. Further research on NDUFA12 variants is essential for a better understanding of their wide phenotypic spectrum.
    Keywords:  Blindness; Saudi Arabia; headache; mitochondrial disease; papilledema
    DOI:  https://doi.org/10.4103/jfcm.jfcm_322_25
  4. Neurol Sci. 2026 Feb 16. 47(3): 264
       BACKGROUND: Mitochondrial disorders are a group of heterogeneous diseases marked by deficiencies in oxidative phosphorylation (OXPHOS). A common subtype, MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes [SLEs]), is primarily linked to variants in mitochondrial transfer RNA (mt-tRNA) genes, yet the molecular mechanisms underlying many of these variants remain poorly understood.
    METHODS: We performed a comprehensive assessment of a 14-year-old male patient, including clinical evaluation, genetic testing, histopathology, and functional biochemical analyses of muscle tissue. A systematic literature review was conducted to compare previously reported MT-TS2 variants and their associated phenotypes.
    RESULTS: We identified a rare m.12244G > A variant in the tRNASer(AGY) gene associated with classical MELAS phenotype. Functional analysis demonstrated impaired mitochondrial translation and OXPHOS dysfunction. Histological findings revealed COX-negative and ragged red fibers, while western blotting indicated downregulation of key mitochondrial proteins. Literature review showed that MT-TS2 variants are associated with variable phenotypes including encephalopathy, myopathy, deafness, diabetes, and retinopathy.
    CONCLUSION: Our study provides the first experimental validation of the pathogenicity of the m.12244G > A variant, confirming its deleterious impact on mitochondrial function. This finding expands the genotype spectrum of MELAS and highlights the importance of functional validation for rare mtDNA variants.
    Keywords:  M.12244G > A variant; MELAS; MT-TS2; Mitochondrial disease; Mitochondrial encephalopathy; SLEs; TRNASer(AGY)
    DOI:  https://doi.org/10.1007/s10072-026-08875-y
  5. Autophagy. 2026 Feb 19. 1-2
      BNIP3L/NIX is a mitophagy receptor highly expressed in the brain. Unlike most mitophagy receptors that are recruited to mitochondria only upon stress, BNIP3L constitutively localizes to the mitochondrial outer membrane, suggesting functions beyond stress-induced mitophagy. Here, we identify a non-mitophagic role of BNIP3L in neuronal physiology. Conditional deletion of Bnip3l in glutamatergic neurons of the basolateral amygdala selectively impairs contextual fear memory in mice, a phenotype rescued by both wild-type BNIP3L and a mitophagy-deficient BNIP3L mutant lacking the LC3-interacting region motif. Mechanistically, BNIP3L competitively binds AMP-activated protein kinase (AMPK), thereby relieving AMPK-dependent inhibitory phosphorylation of DNM1L/DRP1 (dynamin 1 like) at Ser637. This interaction promotes rapid mitochondrial fission, supporting synaptic energy availability during memory encoding. Together, these findings reveal a switchable function of BNIP3L in neurons, acting either to acutely regulate mitochondrial dynamics to meet energetic demand or to engage mitophagy when mitochondrial function becomes compromised.
    Keywords:  BNIP3L/NIX; Basolateral amygdala; fear memory; mitochondrial dynamics; mitophagy
    DOI:  https://doi.org/10.1080/15548627.2026.2634183
  6. Biochemistry (Mosc). 2026 Jan;91(1): 178-187
      L36 is a structural protein of the large ribosomal subunit of bacterial, mitochondrial, and chloroplast ribosomes. L36 stabilizes the peptidyl transferase center and the L7/L12 stalk, which is a binding site for the elongation factors during the translation cycle. According to the cryoelectron microscopy data, L36 incorporates into the large ribosomal subunit in both bacteria and mitochondria at the final assembly step. Bacterial L36 is not an essential protein, since deletion of its gene in bacteria did not impair the colony growth or reduce the mRNA translation levels. Deletion of the RTC6 gene coding for the mitochondrial homologue of L36 (bL36m) in Saccharomyces cerevisiae, impeded yeast growth on the media with non-fermentable carbon sources. Our findings indicate that the mitochondrial dysfunction associated with the absence of bL36m was caused by a decreased activity of cytochrome c oxidase complex that resulted from the selective disruption of synthesis of its subunits encoded in the mitochondrial genome. Furthermore, selective inhibition of mitochondrial protein synthesis did not induce critical structural abnormalities of mitochondrial ribosomes or reduce their ability to bind mRNA. Furthermore, we demonstrated that in contrast to S. cerevisiae, the absence of bL36m protein in human cells had no substantial impact on the synthesis of mitochondrially encoded proteins or mitochondrial ribosome assembly. However, the observed reduction in the mitochondrial respiration in the bL36m-deficient cells may be indicative of disturbances in the respiratory chain organization not associated with the mitochondrial translation.
    Keywords:  mitochondria; mitochondrial translation; regulation of translation
    DOI:  https://doi.org/10.1134/S000629792560348X
  7. bioRxiv. 2026 Feb 12. pii: 2025.12.22.695711. [Epub ahead of print]
      Mitochondrial stress activates the integrated stress response (ISR) through the mitochondrial protein DELE1, which relays stress signals to the cytosolic kinase HRI to induce ATF4. Dysregulation of DELE1-mediated signaling has been implicated in pathological conditions, yet molecular strategies to modulate DELE1 remain unavailable. Here, we report de novo designed proteins that bind DELE1, block its oligomerization, and inhibit DELE1-mediated ISR activation. Several designs form stable complexes with DELE1 and disrupt its oligomerization in vitro while preserving DELE1's ability to bind HRI. In cells, these designs suppress ATF4 induction during mitochondrial stress and impair the recovery of elongated mitochondrial morphology following transient insult. Crystal structure analysis, structural modeling, and targeted mutagenesis confirm that the designed proteins engage a critical interface required for DELE1 oligomerization. These findings establish DELE1 as a druggable target and demonstrate that de novo designed proteins offer precise tools to modulate this pathway, laying groundwork for therapeutic development.
    DOI:  https://doi.org/10.64898/2025.12.22.695711
  8. Sci Adv. 2026 Feb 20. 12(8): eadx7815
      Autosomal dominant optic atrophy (ADOA) is a hereditary optic neuropathy caused by OPA1 variants, leading to retinal ganglion cell (RGC) degeneration and vision loss. The mechanisms behind RGC vulnerability to mitochondrial dysfunction remain unclear. We developed a patient-specific Opa1V291D/+ knock-in mouse model to investigate mitochondrial dysfunction and retinal metabolism in ADOA. We observed that Opa1V291D/+ mice exhibited anatomical and functional RGC abnormalities recapitulating the ADOA phenotypes. Reduced optic atrophy 1 (OPA1) protein levels were noted in Opa1V291D/+ mice, accompanied by decreased protein stability. Moreover, mitochondrial function was compromised, as indicated by reduced Complex I activity, increased oxidative stress, and diminished adenosine triphosphate production in the retinas of Opa1V291D/+ mice. Spatial metabolomics revealed energy deficits in the inner retina and heightened glycolysis in the outer retina. Immunostaining showed decreased expression of glycolytic proteins in the ganglion cell layer. Single-nucleus RNA sequencing disclosed significant down-regulation of energy-production genes in RGCs, while other retinal cell types remained unaffected. These findings emphasize the specific vulnerability of RGCs to bioenergetic crises, connecting disrupted energy homeostasis to their degeneration. By increasing the nicotinamide adenine dinucleotide (NAD+)/reduced form of NAD+ (NADH) redox ratio through the overexpression of mitochondrial-targeted Lactobacillus brevis NADH oxidase (MitoLbNOX) in RGCs, we demonstrated improved RGC function and survival through enhanced energy metabolism and reduced oxidative stress. These findings confirm that disrupted energy metabolism leads to RGC degeneration and emphasize the enhancement of the NAD+/NADH redox ratio as a promising treatment strategy to protect RGCs from degeneration in ADOA.
    DOI:  https://doi.org/10.1126/sciadv.adx7815
  9. STAR Protoc. 2026 Feb 16. pii: S2666-1667(26)00023-7. [Epub ahead of print]7(1): 104370
      Mitochondrial dynamics are essential for cellular homeostasis and can be visualized in adult Drosophila oenocytes using live-cell confocal imaging. Here, we present a protocol for live-cell imaging of mitochondrial dynamics in adult Drosophila oenocytes. We describe steps for fly preparation, dissection of abdominal cuticle to expose oenocytes, and mounting. We then detail procedures for time-lapse acquisition of mitochondria labeled with mitoGFP. Optimized imaging parameters enable reproducible visualization of mitochondrial morphology stably for longer durations.
    Keywords:  Cell Biology; Genetics; Microscopy; Model Organisms
    DOI:  https://doi.org/10.1016/j.xpro.2026.104370
  10. NPJ Genom Med. 2026 Feb 20.
      Mutations in genes encoding mitochondrial proteins are increasingly recognized as a major cause of neurodegenerative disorders, owing to the role of mitochondria in neuronal energy metabolism and signaling. Here, we investigate MTNAP1 (mitochondrial nucleoid-associated protein 1) as a novel gene associated with an autosomal recessive neurodevelopmental disorder characterized by progressive cerebral and cerebellar atrophy. Three affected probands from two unrelated families presented with global developmental delay, ataxia, spasticity, seizures, and progressive neurological decline, with MRI revealing generalized cerebral and cerebellar volume loss and thinning of the corpus callosum. Trio-based exome sequencing uncovered two ultra-rare, biallelic loss-of-function variants in MTNAP1: a homozygous missense variant (p.G553R) in two siblings and a homozygous nonsense variant (p.Y13X) in an unrelated proband. Functional studies in proband-derived fibroblasts and MTNAP1-silenced neuronal cells implicated profound mitochondrial fragmentation, reduced oxidative phosphorylation capacity, increased reactive oxygen species accumulation, and premature senescence-like stress responses. Structural modeling and biophysical analyses revealed that the p.G553R variant destabilizes the MTNAP1 fold, disrupts its DNA- and membrane-binding interfaces, and induces aberrant aggregation, leading to loss of mitochondrial integrity. Collectively, our findings suggest MTNAP1 as a crucial regulator of mitochondrial homeostasis and identify pathogenic MTNAP1 variants as the cause of a novel, progressive neurodegenerative disorder.
    DOI:  https://doi.org/10.1038/s41525-026-00554-3
  11. Proc Natl Acad Sci U S A. 2026 Feb 24. 123(8): e2522583123
      The human long noncoding RNA (lncRNA) RMRP, initially identified as part of the RNase MRP complex, is linked to various human diseases. However, its structural flexibility and broader cellular roles are not well understood. Here, we offer a comprehensive analysis of RMRP's structure in solution, its interactions with human proteins, and its mitochondrial functions. Using small-angle X-ray scattering (SAXS), we show that RMRP adopts different Mg2+-dependent shapes, shifting from an extended Y-shaped form to a more compact one as Mg2+ levels increase. We identified and characterized interactions between RMRP and the DEAD-box RNA helicases DDX5 and DDX3X, with DDX5 binding strongly and exhibiting ATP-dependent helicase activity on RMRP, while DDX3X mainly acts as an expression regulator. Both helicases are crucial for the proper mitochondrial localization of RMRP, working within a complex regulatory network. Functionally, reducing RMRP levels disrupts mitochondrial stability, leading to membrane depolarization and an increase in reactive oxygen species, without affecting cell growth. Mechanistically, RMRP specifically controls nuclear-encoded mitochondrial proteins involved in cristae structure (DNAJC11) and respiratory chain function (NDUFS8). Our results position RMRP as a structurally adaptable lncRNA that collaborates with RNA helicases to preserve mitochondrial health through specific gene regulation. These insights provide perspectives on RMRP's biology and the molecular mechanisms underlying RMRP-related disorders, which could inform future therapies for conditions resulting from RMRP dysfunction.
    Keywords:  DEAD-box helicases; RMRP; long noncoding RNA; mitochondrial function
    DOI:  https://doi.org/10.1073/pnas.2522583123
  12. FEBS J. 2026 Feb 18.
      Brown adipose tissue (BAT) plays a central role in thermogenesis by coupling fatty acid oxidation to heat production. Efficient BAT thermogenic activity requires enhanced glycolytic flux, which in turn depends on continuous regeneration of cytosolic NAD+ to sustain glyceraldehyde-3-phosphate dehydrogenase activity. This regeneration is mediated by three main pathways: lactate dehydrogenase, the glycerol-3-phosphate shuttle (GPSh), and the malate-aspartate shuttle (MASh). We previously showed that inhibition of the mitochondrial pyruvate carrier increases energy expenditure in brown adipocytes via MASh activation. However, the specific contribution of MASh to BAT energy metabolism remains poorly defined. Here, we show that MASh is functional and directly regulates lipid metabolism in BAT. Enzymatic activities of cytosolic and mitochondrial malate dehydrogenases and glutamic-oxaloacetic transaminases in BAT were comparable to those in the liver. Using a reconstituted system of isolated BAT mitochondria and cytosolic MASh enzymes, we demonstrated that extra-mitochondrial NADH is efficiently reoxidized in a glutamate-dependent manner via MASh. Genetic silencing of the mitochondrial carriers critical to MASh, namely the oxoglutarate carrier (Ogc) and aspartate-glutamate carrier (Aralar1), had no apparent effects on respiratory rates. However, silencing either Ogc or Aralar1 led to the accumulation of small lipid droplets and impaired norepinephrine-induced lipolysis. Taken together, our data indicate a novel role of MASh in regulating BAT lipid homeostasis with potential implications to body energy expenditure and thermogenesis.
    Keywords:  energy; heat; metabolism; obesity; redox; thermogenesis
    DOI:  https://doi.org/10.1111/febs.70461
  13. J Child Neurol. 2026 Feb 18. 8830738261420613
      
    Keywords:  ADEM; encephalitis; encephalomyeloradiculoneuropathy; lymphoma; mitochondrial disorder
    DOI:  https://doi.org/10.1177/08830738261420613
  14. Commun Biol. 2026 Feb 14.
      Genetic pain loss disorders represent a heterogeneous group of rare diseases mainly characterized by defective nociception. Understanding the underlying molecular mechanism is fundamental to improve the treatment of patients affected by these rare disorders. Feline Leukemia Virus Subgroup C Receptor 1 (FLVCR1) is one of the genes previously associated with sensory neuropathy that requires further investigation. Here, we report on two additional patients with novel disease-causing variants in FLVCR1 and introduce a zebrafish model of the disease. The analyses of patient-derived fibroblasts show that distinct FLVCR1 variants compromised all the known functions associated with FLVCR1, thus affecting choline levels, heme biosynthesis and mitochondrial Ca2+ handling. Furthermore, we provide evidence that the alteration of these processes impairs the TCA cycle and OXPHOS, and induces lipid peroxidation. Our data points to the alterations of energetic metabolism as a potential driving pathomechanism in FLVCR1-associated sensory neuropathy.
    DOI:  https://doi.org/10.1038/s42003-026-09691-y
  15. J Cell Sci. 2026 Feb 19. pii: jcs.264304. [Epub ahead of print]
      ADP-ribosylation factor-like 4D (Arl4D), a Ras small GTPases superfamily member, plays critical roles in membrane trafficking, cytoskeletal remodeling, and cell migration. GDP-bound Arl4D was discovered previously to locate at the mitochondria and alter mitochondrial morphology and activity; however, how the nucleotide binding state and mitochondrial targeting of Arl4D is regulated had remained unclear. We now discover that TBC1D15, a well-known Rab7 GTPase-activating protein (GAP), functions also as an Arl4D GAP to promote Arl4D mitochondrial targeting. We initially find that GDP-bound Arl4D translocates to the mitochondria under serum starvation and affects mitochondrial homeostasis. We then find that TBC1D15 interacts with Arl4D through the TBC domain and promotes GTP hydrolysis of Arl4D. Knockdown of TBC1D15 leads to an increase in Arl4D activity and decreased Arl4D mitochondrial translocation under serum starvation. These findings support that TBC1D15 acts as an Arl4D GAP and reveal a new role for this GAP in modulating mitochondrial homeostasis.
    Keywords:  ADP-ribosylation factor; Arl4; GTPase; GTPase activating protein; Mitochondria
    DOI:  https://doi.org/10.1242/jcs.264304
  16. J Biochem. 2026 Feb 20. pii: mvag014. [Epub ahead of print]
      In mitochondria, the pyruvate dehydrogenase complex (PDHC) serves as a key metabolic regulator by converting glycolysis-derived pyruvate into acetyl-CoA, thereby controlling carbon flux into the tricarboxylic acid (TCA) cycle. PDHC activity is tightly regulated by two post-translational modifications: phosphorylation of the E1 subunit and lipoylation of the E2 subunit. While phosphorylation of E1 reversibly suppresses pyruvate dehydrogenase (PDH) activity, lipoylation of E2 is essential for intracomplex electron transfer reactions, and together these modifications define PDHC enzymatic activity. Mitochondrial respiratory supercomplexes (SCs) play a critical role in efficient electron transfer during mitochondrial respiration, and PDH has been reported to regulate SC organization. However, it remains unclear whether this regulatory mechanism, including subunit phosphorylation, is linked to protein lipoylation. In this study, we examined the impact of protein lipoylation on the phosphorylation status of the PDHC E1 subunit and on mitochondrial respiratory supercomplex formation during C2C12 differentiation. To this end, suppression of lipoic acid synthase (LIAS), a key enzyme responsible for mitochondrial protein lipoylation, in C2C12 cells resulted in dephosphorylation of the PDHC E1 subunit and formation of specific mitochondrial respiratory supercomplexes. These findings suggest that PDHC E1 dephosphorylation and specific mitochondrial respiratory supercomplex assembly can occur under conditions of impaired E2 lipoylation.
    Keywords:  C2C12 differentiation; LIAS; Mitochondria; PDHC; Respiratory supercomplexes
    DOI:  https://doi.org/10.1093/jb/mvag014
  17. Mitochondrion. 2026 Feb 17. pii: S1567-7249(26)00026-7. [Epub ahead of print] 102136
      Sideroflexin 4 (SFXN4) is a transmembrane protein located in the inner membrane of the mitochondria. SFXN4 is also thought to be involved in the formation of iron-sulphur centres. Deleterious bi-allelic variants of the SFXN4 gene have been reported in only 3 patients, with a phenotype including intellectual disability and macrocytic anaemia. We describe here a patient carrying pathogenic variants of SFXN4, associated with a non-anaemic sideroblastic macrocytosis and a complex I deficiency.
    Keywords:  Macrocytosis; Mitochondrial disease; SFXN4
    DOI:  https://doi.org/10.1016/j.mito.2026.102136
  18. FASEB J. 2026 Feb 28. 40(4): e71603
      Aggregation of TDP-43 in neuronal cells is a defining neuropathological hallmark of amyotrophic lateral sclerosis (ALS). Emerging evidence suggests that TDP-43 pathology also occurs in skeletal muscle fibers, but its functional significance in myocytes remains poorly understood. In this study, we utilized the C2C12 myoblast cell to investigate the subcellular localization of TDP-43 during myogenic differentiation. Our findings demonstrate that TDP-43 progressively translocates to mitochondria in parallel with myotube maturation. Notably, increased mitochondrial localization of TDP-43 was also observed in skeletal muscle tissues from patients with ALS, corroborating the clinical relevance of this phenomenon. Functional assays revealed that inhibition of TDP-43 mitochondrial translocation significantly enhances myotube maturation. Collectively, these results support a pathophysiological role for aberrant mitochondrial mislocalization of TDP-43 in regulating myogenic differentiation and contributing to muscle degeneration in TDP-43 proteinopathies.
    Keywords:  TDP‐43; amyotrophic lateral sclerosis; mitochondria; myotube maturation
    DOI:  https://doi.org/10.1096/fj.202504624R
  19. Autophagy Rep. 2026 ;5(1): 2627062
      In PINK1 (PTEN induced kinase 1)/PRKN (Parkin)-mediated mitophagy, the rupture of the outer mitochondrial membrane (OMM) emerges as a crucial event required for efficient mitochondrial clearance. Mechanistically, OMM rupture exposes inner mitochondrial membrane (IMM) mitophagy receptors, facilitating subsequent autophagic removal. Despite the important role of OMM rupture in mitophagy, the underlying mechanism remains elusive and technically difficult to monitor. In a recent study, we developed a novel fluorescent biosensor to directly visualize OMM rupture. This technique enables temporal and spatial characterization of OMM rupture and provides a powerful platform to dissect the underlying mechanism. Using this tool, we revealed that VCP (valosin containing protein) and its recruitment factors are required for OMM rupture, suggesting that VCP-dependent remodeling of the OMM proteome primes the rupture of OMM during mitophagy. Abbreviations: ARIH1, Ariadne RBR E3 ubiquitin protein Ligase 1; AMFR, autocrine motility factor receptor; ANKRD13A, ankyrin repeat domain-containing protein 13 A; FUNDC1, FUN14 domain containing 1; OA, oligomycin and antimycin; CID, chemical-induced dimerization; IMM, nner mitochondrial membrane; LC3, microtubule-associated protein 1 light chain 3; MUL1, mitochondrial E3 ubiquitin protein ligase 1; NIX, BCL2 interacting protein 3 like; OMM, outer mitochondrial membrane; UBXN1, ubiquitin regulatory X domain-containing protein 1; UBXN6, ubiquitin regulatory X domain-containing protein 6; VCP, valosin-containing protein; WIPI2, WD repeat domain phosphoinositide interacting protein 2.
    Keywords:  Biosensor; Mitochondrial outer membrane rupture; Mitochondrial quality control; PINK1/Parkin-mediated mitophagy; VCP
    DOI:  https://doi.org/10.1080/27694127.2026.2627062
  20. Mitochondrion. 2026 Feb 18. pii: S1567-7249(26)00025-5. [Epub ahead of print] 102135
      Aminoacyl-tRNA synthetases (aaRSs) are multi-domain enzymes that, in addition to their catalytic and tRNA-anticodon-binding domains, may include clade-specific extra regions conferring unique properties. These extra domains are poorly characterized in mitochondrial aaRSs (ARS2), complicating genetic diagnosis. ARS2 enzymes are essential for mitochondrial translation, broadly expressed, and pathogenic variants in any domain can cause varied neurological disorders. Here, we show how diagnosis challenge leads to a fundamental discovery. PARS2 deficiency, caused by pathogenic variantsin the nuclear gene encoding mitochondrial prolyl-tRNA synthetase (ProRS), was reported in few patients. Here, we describe two unrelated patients with epileptic encephalopathy who carry biallelic PARS2 variants including one novel variant predicted as "likely benign" due to poor interspecies conservation of the impacted region.First, through thorough phenotypic evaluation, we confirmed that both patients' clinical features match those of a cohort of 22 patients previously reported with PARS2 deficiency. Next, using comparative protein-structure modeling and a detailed clade-specific analysis of sequence conservation, we discovered that this variant actually falls within a previously unrecognized zinc-binding domain (ZBD), structurally similar to the well-characterized and essential ZBD found in cytosolic ProRS. Our findings underscore the limitations of existing tools for predicting the pathogenicity of ARS2 variants and demonstrate the value of integrating structural modeling with evolutionary conservation analysis. In conclusion, this work not only reveals a critical ZBD in PARS2, offering new insights into its structural and functional properties but also expands the genotypic spectrum of PARS2-related disorders and provides a comprehensive description of the associated phenotypes.
    Keywords:  Aminoacyl-tRNA synthetases; Epileptic encephalopathy; PARS2; Zinc binding domain
    DOI:  https://doi.org/10.1016/j.mito.2026.102135
  21. Diabetes Obes Metab. 2026 Feb 19.
       AIMS: Growth differentiation factor 15 (GDF15) has emerged as a promising metabolic regulator with hepatoprotective properties in metabolic dysfunction-associated steatotic liver disease (MASLD), yet its underlying mechanisms remain elusive. Given that mitochondria are the primary site of fatty acid oxidation (FAO) and that mitochondrial morphodynamics are critical for normal hepatic lipid metabolism, we investigated how GDF15 regulates hepatic lipid homeostasis through mitochondrial dynamics.
    MATERIALS AND METHODS: We established cellular steatosis models using primary rat hepatocytes exposed to lipotoxic palmitate (PA) or non-lipotoxic free fatty acid mixture (FFA, oleate: palmitate = 2: 1). Following GDF15 administration, we quantified lipid droplet content, expression of lipid metabolism genes, mitochondrial fatty acid translocation, and mitochondrial morphodynamics and function. The mechanistic role of ERK1/2 signalling was assessed through pharmacological inhibition. These findings were subsequently validated in adult progenitor cell-derived human liver organoids.
    RESULTS: GDF15 significantly mitigated both PA- and FFA-induced lipid accumulation by upregulating key FAO genes and down regulating lipid synthesis genes. Importantly, GDF15 corrected PA-induced mitochondrial fusion-fission imbalance by increasing mitochondrial fusion proteins MFN1 and OPA1 while modulating the activation of fission regulator DRP1. GDF15 enhanced fatty acid translocation into mitochondria and improved FAO. Mechanistically, GDF15 exerted these effects partially through inhibition of the ERK1/2 signalling pathway. Human liver organoid models further corroborated this protective mechanism of GDF15 against hepatic steatosis.
    CONCLUSIONS: Our study reveals that, specifically under lipotoxic conditions, GDF15 alleviates hepatocyte steatosis by preserving mitochondrial morphodynamics homeostasis and enhancing mitochondrial FAO capacity via ERK1/2 inhibition. These condition-specific mechanisms provide critical insights into GDF15's hepatoprotective effects and support its further investigation as a potential therapeutic target for MASLD.
    Keywords:  GDF15; MASLD; lipid metabolism; mitochondrial morphodynamics
    DOI:  https://doi.org/10.1111/dom.70561
  22. PLoS One. 2026 ;21(2): e0343604
      Age-associated declines in skeletal muscle function are linked to cellular senescence and mitochondrial alterations, yet mitochondrial phenotypes in aged human myoblasts remain insufficiently characterized. Here, we examined primary skeletal muscle myoblasts from young and elderly donors to assess mitochondrial function, morphology, and mitochondria-endoplasmic reticulum (ER) contact sites (MERCS). Myoblasts from older donors exhibited senescence features, including elevated SA-β-gal activity and reduced Lamin B1 expression, accompanied by increased mitochondrial oxidative stress. Despite marked mitochondrial hyperfusion and increased mitochondrial DNA content, mitochondrial oxygen consumption rate and membrane potential per mitochondrial area were comparable between young and old cells. MERCS were significantly elevated in aged myoblasts and were reduced by scavenging mitochondrial reactive oxygen species (mtROS), indicating an association between oxidative stress and MERCS formation. These findings suggest that mitochondrial hyperfusion and enhanced MERCS accompany cellular aging in human myoblasts and may contribute to maintaining mitochondrial function under elevated oxidative stress.
    DOI:  https://doi.org/10.1371/journal.pone.0343604
  23. Mol Biol Evol. 2026 Feb 17. pii: msag044. [Epub ahead of print]
      Mitochondria are essential for cellular energy production and biosynthesis, thermogenesis, and cell signalling, and thus help coordinate physiological responses to changing environments. Humans (Homo sapiens) have adapted to cope with a wide range of climatic conditions, however the role of the mitochondrial genome (mtDNA) in mediating this process remains poorly understood. Here we curated a dataset of 19,570 publicly available full human mitochondrial genomes, an approximate 40-fold increase on earlier studies, paired with modern climate and reconstructed paleoclimate variables. Using a Generalised Linear Model approach, we identify independent candidate variants significantly associated with climatic conditions, suggesting local adaptation in human mitochondrial genomes. Candidate variants are distributed across multiple loci in regulatory, tRNA, rRNA and protein-coding regions - including prominently in ND2 and ND4 complex I subunits. Specific variants are predicted to impact mtDNA transcription, ribosome or protein structure, and multiple have been associated with disease pathologies. We further show that candidate variant genotype distributions are each best modelled by different paleo-bioclimatic variables, consistent with environmental stressors linked to our measured variables exerting subtly distinct selective effects. These stressors may reflect dietary changes or different thermogenic demands at lower temperatures. Our results provide genetic evidence to support the accumulating body of work from functional studies that mitochondria can modulate adaptation to diverse environments. This work underscores the importance of mtDNA in evolutionary biology and its relevance for understanding both disease and physiological variation in global populations.
    Keywords:   Homo sapiens ; Mitochondria; adaptation; climate; humans; mitochondrial genome; mtDNA; natural selection; rRNA; tRNA
    DOI:  https://doi.org/10.1093/molbev/msag044
  24. J Cell Physiol. 2026 Feb;241(2): e70153
      Human embryonic stem cells (hESCs) hold immense promises for regenerative medicine and exhibit two distinct pluripotency states: primed and naïve. However, metabolic regulation underlying these states remains incompletely understood. In particular, mitochondrial pyruvate oxidation in pluripotency regulation has not been documented. Here, we combined an inducible dihydrolipoamide S-acetyltransferase (DLAT) knockout model and pharmacological inhibition of mitochondrial pyruvate uptake (via the mitochondrial pyruvate carrier inhibitor UK5099) to dissect the state-specific effects of mitochondrial pyruvate oxidation in isogenic naïve and primed hESCs. Primed hESCs lacking DLAT or treated with UK5099 displayed pronounced cell death, reduced global protein acetylation levels, and transcriptional dysregulation. These defects were partially rescued by sodium acetate supplementation, implicating a reduction in acetyl-CoA abundance as a key mechanism. Notably, a set of neural lineage genes was specifically downregulated by disrupted mitochondrial pyruvate oxidation in primed hESCs, revealing the importance of mitochondrial pyruvate oxidation-mediated acetyl-CoA production in priming neural differentiation. In line with this, disruption of mitochondrial pyruvate oxidation impaired the differentiation process of primed hESCs towards neuroectoderm. In contrast, DLAT depletion in naïve hESCs did not affect cell growth and the naïve pluripotency state, highlighting the pluripotency state-dependent function of mitochondrial pyruvate oxidation. Our study uncovers the pivotal roles of mitochondrial pyruvate oxidation-mediated acetyl-CoA production for sustaining survival and transcriptional fidelity as well as facilitating neural differentiation in primed hESCs. Moreover, we emphasize that the function of mitochondrial pyruvate oxidation in hESCs is pluripotency state-dependent. These findings provide new cues for optimizing hESC maintenance and differentiation through targeted metabolic manipulation.
    Keywords:  DLAT; acetyl‐CoA; histone acetylation; human embryonic stem cells; human naïve pluripotency; human primed pluripotency; mitochondrial pyruvate oxidation
    DOI:  https://doi.org/10.1002/jcp.70153
  25. Nature. 2026 Feb 18.
      
    Keywords:  Genomics; Machine learning; Medical research
    DOI:  https://doi.org/10.1038/d41586-026-00290-9
  26. Plant Cell Physiol. 2026 Feb 19. pii: pcag006. [Epub ahead of print]
      The FTSH4 protease is a major component of the Arabidopsis mitochondrial protein quality control system. It has both a proteolytic and a chaperone-like activity and forms complexes anchored in the inner mitochondrial membrane. Here, we show that FTSH4 assembles into two distinct forms: a dominant high-molecular-weight megacomplex with stomatin-like protein 1 (SLP1), and smaller SLP1-free assemblies. In the slp1-1 mutant, the FTSH4-SLP1 megacomplex is absent, while the abundance of SLP1-free FTSH4 assemblies is nearly doubled. Despite this, slp1-1 maintains wild-type levels of FTSH4 substrates, TIM17-2 and NAD9, indicating that the SLP1-free assemblies retain proteolytic activity. Furthermore, slp1-1 mitochondria accumulate fewer detergent-resistant HSP23.6 aggregates under elevated temperature than ftsh4-1 and even wild-type. Consequently, the mitochondrial unfolded protein response reported in ftsh4-1 is not induced in slp1-1. Although slp1-1 plants display morphological changes previously associated with ftsh4-1, such as shorter inflorescence stems due to premature arrest of the shoot apical meristem, these are less pronounced. Taken together, the increased abundance of SLP1-free FTSH4 assemblies is sufficient to support general mitochondrial proteostasis, providing effective protection against heat-induced aggregation of mitochondrial proteins. In contrast, the FTSH4-SLP1 megacomplex more effectively fulfils the meristem-specific functions of FTSH4.
    Keywords:   Arabidopsis thaliana ; FTSH4 protease; SLP1; mitochondria; protein complexes; stomatin
    DOI:  https://doi.org/10.1093/pcp/pcag006
  27. Bioinformatics. 2026 Feb 19. pii: btag086. [Epub ahead of print]
       MOTIVATION: Long-read sequencing (LRS) is increasingly used for human medical research and clinical diagnostics, due to its capacity to generate complete genome information. However, there is a lack of robust and easy-to-use pipelines for comprehensive LRS data analysis.
    RESULTS: Here we present Nallo, a Nextflow pipeline for analysis of PacBio and Oxford Nanopore data, with additional support for rare disease research projects. The pipeline detects a wide range of genetic variants, performs genome assembly and reports CpG methylation. It also enables annotation and ranking of variants based on their predicted functional consequences.
    AVAILABILITY: Nallo is available from GitHub: https://github.com/genomic-medicine-sweden/nallo.
    DOI:  https://doi.org/10.1093/bioinformatics/btag086
  28. Adv Sci (Weinh). 2026 Feb 17. e02239
      Ferroptosis is a type of regulated cell death characterized by the accumulation of lipid peroxides that damage cell membranes specifically. Mitochondrial swelling and dysfunction are hallmarks of ferroptosis; however, what causes mitochondrial swelling and the consequences of mitochondrial swelling in ferroptotic signal transduction remain poorly understood. Our study found that mitochondrial permeability transition pore (mPTP) opening is essential for mitochondrial swelling and ferroptosis activation. During ferroptosis, oxidized mitochondrial DNAs (mtDNAs) are released through the mPTP. These oxidized mtDNAs activate the cyclic GMP-AMP synthase (cGAS)-stimulator of interferon genes (STING) pathway, promoting ferroptosis through activating ferrotinophagy. Consistently, inhibition of mtDNA-repair enhances cellular sensitivity to ferroptosis and therefore synergizes with ferroptosis inducer in suppressing tumorigenesis in mouse xenograft tumor models. This study provides a fundamental understanding of how mPTP engages in ferroptosis by releasing mitochondrial DNAs as crucial messengers to activate ferroptotic signaling.
    Keywords:  cGAS‐STING; ferroptosis; mPTP; mitochondria; mtDNA
    DOI:  https://doi.org/10.1002/advs.202502239
  29. Mol Genet Metab. 2026 Feb 12. pii: S1096-7192(26)00035-1. [Epub ahead of print]147(4): 109752
       BACKGROUND: CLPB-related mitochondrial disease causes congenital neutropenia, developmental delay/intellectual disability, progressive brain atrophy, movement disorders, cataracts, and 3-methylglutaconic aciduria. Both monoallelic and biallelic forms exist. This retrospective cohort study compared clinical outcomes and genotype-structure-phenotype correlations across zygosity groups.
    METHODS: Sixty-three individuals (41 biallelic, 22 monoallelic; 6 unpublished) with disease-causing CLPB variants were identified via literature review and a multicenter survey. In silico modeling assessed structural impact. A modified CLPB Disease Burden Index (DBI) quantified severity.
    RESULTS: Median age at last follow-up was 4.0 years (IQR: 0.25-12.6) in biallelic and 12.0 years (IQR: 5.3-21.0) in monoallelic cases. Death occurred in 66% of biallelic and 23% of monoallelic individuals, with earlier median age at death in biallelic cases (6 months vs 2.4 years). Biallelic cases had significantly higher DBI scores and poorer survival (4-year survival: 50% vs 82%). Stop/stop genotypes were associated with greater disease burden than missense combinations. Structural predictions-particularly variants causing nonsense-mediated decay or ankyrin domain disruption-were stronger survival predictors than zygosity or age of onset. Early-onset disease (<12 months) correlated with more severe progression. Later onset often resulted in milder phenotypes. Hematologic and neurologic features overlapped across zygosity; cataracts and dystonia were more common in biallelic cases. Milestone attainment was poor, with <50% walking or speaking, and only 10-20% doing so on time. Four monoallelic patients received hematopoietic stem cell transplants with mixed outcomes. Granulocyte colony-stimulating factor was associated with improved survival.
    CONCLUSIONS: This is the largest cohort study to date comparing biallelic and monoallelic CLPB deficiency. Structural variant impact-particularly ankyrin domain disruption-emerged as a key prognostic factor.
    Keywords:  3-methylglutaconic aciduria; Ankyrin repeat region; CLPB; Cataracts; Congenital neutropenia; Genotype-phenotype correlation; Mitochondrial chaperonopathy; Protein modeling; Zygosity
    DOI:  https://doi.org/10.1016/j.ymgme.2026.109752
  30. Cell Res. 2026 Feb 16.
      Aging is the primary cause of cognitive decline. Despite extensive study, the molecular mechanisms driving aging-associated cognitive decline remain unclear. Here, we describe a proteostasis-independent function of SEC61A1 and its involvement in aging-associated cognitive decline. SEC61A1 regulates ER-mitochondria contact sites, affecting mitochondrial DNA and RNA synthesis and subsequently leading to changes in innate immune signaling mediated by mitochondrial double-stranded RNA (mt-dsRNA). This pathway is activated in aged wild-type mice, Alzheimer's disease patients, and 5×FAD mice. Tissue-specific overexpression of Sec61a1 in the mouse cortex (Sec61a1Tg) is sufficient to induce cognitive decline without affecting motor activity. Knockdown of Sec61a1 or Mavs ablates mt-dsRNA-mediated innate immune signaling and alleviates cognitive decline in naturally aging wild-type mice. These results reveal a molecular mechanism of aging- and disease-associated cognitive decline and provide a potential therapeutic tool for intervention.
    DOI:  https://doi.org/10.1038/s41422-026-01224-w
  31. Adv Sci (Weinh). 2026 Feb 17. e19792
      Metastatic invasiveness emerges from coordinated intrinsic programs and microenvironmental cues that converge on mitochondrial quality control (MQC). Here, we use "context" to denote stage- and site-aware constellations of tumor-intrinsic states (e.g., mtROS tone, mtDNA integrity, epigenetic wiring, cellular stiffness, oncogenic mutations) and extrinsic landscapes (oxygen-nutrient availability, ECM mechanics, stromal/inflammatory signals). These axes jointly shape mitochondrial adaptation by tuning bioenergetics, redox balance, metabolic plasticity, fission-fusion dynamics, mechanosensitive hubs, and Ca2 + homeostasis. As pressures intensify, mitochondrial vulnerabilities-such as mtDNA compromise and mtUPR activation-signal the engagement of mitophagy to preserve organelle fitness under stress. Through these coupled changes in mitochondrial performance and stress responses, context governs EMT/MET plasticity and transitions across migratory, invasive, and proliferative states. Mechanistically, ubiquitin conjugation, via E3 ligases and deubiquitinases, serves as an integrating conduit that links mitochondrial remodeling and mitophagy to cytoskeletal reprogramming and invasive behavior. This ubiquitin-mitochondria interface therefore represents a coherent therapeutic entry point; translational strategies including PROTAC-enabled targeting and selective E3/DUB or mitophagy-pathway modulators may rebalance pathological ubiquitin signaling, restore mitochondrial homeostasis, and constrain tumor dissemination.
    Keywords:  EMT–MET plasticity; extracellular matrix mechanics; hypoxia and nutrient deprivation; mitochondrial ROS; mitochondrial dynamics; mitophagy; ubiquitination
    DOI:  https://doi.org/10.1002/advs.202519792
  32. ACS Med Chem Lett. 2026 Feb 12. 17(2): 298-299
      Provided herein are novel acrylamide derivatives as mitochondrial permeability transition pore (mPTP) inhibitors, pharmaceutical compositions, use of such compounds in treating neurodegenerative diseases, and processes for preparing such compounds.
    DOI:  https://doi.org/10.1021/acsmedchemlett.5c00778
  33. Proc Natl Acad Sci U S A. 2026 Feb 24. 123(8): e2532796123
      The integrated stress response (ISR) plays a crucial role in cognition via bidirectional modulation of the two major forms of synaptic plasticity, long-term potentiation, and long-term depression (LTD). Specifically, inhibition of the ISR blocks metabotropic glutamate receptor-dependent LTD (mGluR-LTD), whereas its activation facilitates this form of synaptic depression. However, the contribution of activating transcription factor 4 (ATF4), the best studied downstream effector of the ISR, to mGluR-LTD remains unknown. Here, we show that pharmacological activation of group I mGluRs in mouse hippocampal slices increases ATF4 protein levels without altering its transcription and concurrently downregulates the expression of oxidative phosphorylation (OXPHOS) proteins. Selective deletion of ATF4 in excitatory neurons impairs mGluR-LTD and prevents the downregulation of OXPHOS proteins. Notably, administration of a small molecule inhibitor of OXPHOS rescues the impaired mGluR-LTD in ATF4-depleted hippocampal slices, indicating that ATF4 regulates this type of synaptic plasticity by modulating mitochondrial function. Moreover, ATF4 deletion in excitatory neurons disrupts object-place learning, an mGluR-LTD-dependent behavior paradigm. Together, these findings reveal a role of ATF4 as a key mediator of protein synthesis-regulated synaptic depression and related behaviors.
    Keywords:  integrated stress response; mGluR-LTD; synaptic plasticity
    DOI:  https://doi.org/10.1073/pnas.2532796123
  34. Nat Commun. 2026 Feb 17.
      Cell fate determination is closely linked to metabolic state, yet how metabolic remodeling influences human pluripotent stem cells differentiation into three germ layers remains incompletely understood. Here, we reveal that definitive endoderm differentiation from human pluripotent stem cells requires a TGFβ-driven metabolic switch characterized by reduced lactate production and enhanced TCA cycle activity and oxidative phosphorylation, mediated by PDHB. Disruption of glucose utilization or pyruvate entry into the TCA cycle markedly impairs endoderm differentiation, whereas inhibition of lactate production enhances differentiation efficiency. Mechanistically, blockade of glucose metabolism or the TCA cycle reduces intracellular ATP levels, compromising the activity of BAF complex, an ATP-dependent chromatin remodeling complex centered on BRG1. This complex promotes chromatin accessibility and activates endodermal gene programs during differentiation. Together, these findings highlight metabolic reprogramming as a key regulator of human endoderm fate through ATP-dependent control of chromatin remodeling.
    DOI:  https://doi.org/10.1038/s41467-026-69510-0
  35. Nat Neurosci. 2026 Feb 16.
      Understanding the development of midbrain dopaminergic (mesDA) neurons is essential for advancing cell replacement therapies for Parkinson's disease. In the developing ventral midbrain (VM), radial glia (Rgl) cells are the progenitors of mesDA neurons. However, distinct Rgl subtypes have recently been identified, and their individual roles are unclear. Here we analyze transcriptomic data from mouse and human VM Rgl to define their contributions to mesDA neuron development. We identify Rgl1 as the progenitor of the mesDA lineage, and reveal a Rgl1 transcriptional network coordinated by BMAL1, which we validate as a new regulator of mesDA neurogenesis. Moreover, we uncover Rgl3 as a key signaling subtype and show that factors expressed by Rgl3 promote the survival and yield of human stem cell-derived mesDA neurons. Our findings delineate distinct roles of Rgl subtypes, elucidate lineage relationships in the developing VM and uncover new factors that improve the derivation of clinically relevant human mesDA neurons.
    DOI:  https://doi.org/10.1038/s41593-026-02200-8
  36. Mol Cell. 2026 Feb 13. pii: S1097-2765(26)00032-8. [Epub ahead of print]
      Inter-organellar communication is critical for cellular metabolism. One of the most abundant inter-organellar interactions occurs at the endoplasmic reticulum and mitochondria contact sites (ERMCSs). However, an understanding of the mechanisms governing ERMCS regulation and their roles in cellular metabolism is limited by a lack of tools that permit temporal induction and reversal. Through screening approaches, we identified fedratinib, an FDA-approved drug that dramatically increases ERMCS abundance by inhibiting the epigenetic modifier BRD4. Fedratinib rapidly and reversibly modulates mitochondrial and ER morphology, induces a distinct ER-mitochondria envelopment structure, and alters metabolic homeostasis. Moreover, ERMCS modulation depends on mitochondrial electron transport chain complex III function. Comparison of fedratinib activity to other reported inducers of ERMCSs revealed common mechanisms of induction and function, providing clarity to a growing body of experimental observations. In total, our results uncovered a novel epigenetic signaling pathway and an endogenous metabolic regulator that connects ERMCSs and cellular metabolism.
    Keywords:  bromodomain protein; endoplasmic reticulum-mitochondria contact sites; high-throughput screening; mitochondrial electron transport chain
    DOI:  https://doi.org/10.1016/j.molcel.2026.01.012
  37. Nat Cell Biol. 2026 Feb 20.
      Lipid metabolism has recently regained considerable attention in neuroscience, as disturbances in lipid metabolic pathways have been linked to neurodevelopmental and neurodegenerative diseases. Here we examine brain lipid metabolism from a cellular perspective, focusing on lipid uptake, de novo synthesis, storage, breakdown and intercellular transfer. We cover the recent literature showing how these processes are important during brain development and how they occur in diverse brain cell types, including astrocytes, oligodendrocytes, neural stem and progenitor cells, microglia and neurons in the adult brain. We further discuss the consequences of disrupted lipid metabolism and highlight emerging insights into neuron-glia lipid exchange, as well as the importance of lipid droplets for brain health and disease.
    DOI:  https://doi.org/10.1038/s41556-026-01880-5
  38. Sci Rep. 2026 Feb 19.
      
    Keywords:  6-Hydroxydopamine (6-OHDA); Mitochondrial dysfunction; Neurodegenerative diseases; Neuroprotection; Oxidative stress; URG7 protein
    DOI:  https://doi.org/10.1038/s41598-026-38925-6
  39. Curr Opin Cell Biol. 2026 Feb 19. pii: S0955-0674(26)00012-8. [Epub ahead of print]99 102624
      The most common, high-risk genetic factors for Parkinson's disease are pathogenic LRRK2 variants that increase LRRK2 kinase activity and pathogenic GBA1 variants that reduce lysosomal glucocerebrosidase activity. LRRK2 phosphorylates a subset of Rab GTPases, enabling them to bind phosphorylation-specific effectors that drive cellular pathology. To date, LRRK2 has at least two major cellular roles: it promotes exocytosis of lysosome-related organelles-particularly under conditions of lysosome stress in macrophages and microglia-and it regulates the formation and stabilization of primary cilia in neurons and astrocytes. In the brain, loss of primary cilia or GBA1 deficiency impairs Hedgehog signaling, reducing production of neuroprotective factors needed to support vulnerable dopamine neurons. Remarkably, administration of a LRRK2 inhibitor to LRRK2 mutant mice restores cilia and rescues neuroprotective factor production, offering great promise for people with Parkinson's.
    DOI:  https://doi.org/10.1016/j.ceb.2026.102624
  40. Nat Commun. 2026 Feb 18.
      Chronic lymphocytic leukemia (CLL) can arise from lymphoid clonal hematopoiesis of indeterminate potential (L-CHIP), but many individuals who develop CLL lack detectable L-CHIP prior to diagnosis. To identify additional predictors of CLL risk, we analyze mitochondrial heteroplasmy in 419,154 individuals from the UK Biobank (UKB). Heteroplasmy is associated with a 1.5-fold increased risk of developing CLL, and this risk rises to 4-fold when accounting for deleterious heteroplasmic variants. These findings are confirmed in an independent cohort, the All of Us Research Program (AoU). Notably, the associations remain significant even in the absence of L-CHIP, highlighting heteroplasmy's potential utility as an independent biomarker. Moreover, heteroplasmy is enriched in individuals with high-risk L-CHIP genotypes and large clonal burden, suggesting a potential biological role in malignant transformation. Here, we show that mitochondrial heteroplasmy, especially functionally deleterious variants, identifies individuals at increased risk of CLL who would otherwise go undetected by L-CHIP-based assessments.
    DOI:  https://doi.org/10.1038/s41467-026-69861-8
  41. Mol Genet Metab Rep. 2026 Mar;46 101296
       Background: Mutations in COQ8A cause primary coenzyme Q10 deficiency, which can present clinically heterogeneously: Symptoms range from cerebellar ataxia, epilepsy, encephalomyopathy, macular degeneration to nephropathy. High-dose coenzyme Q10 supplementation is widely used, yet there is little evidence on complementary strategies, particularly for non-epileptic features such as cerebellar ataxia.
    Case presentation: We report a 46-year-old female with genetically confirmed COQ8A-related coenzyme Q10 (CoQ10) deficiency, presenting with ataxia and epilepsy characterized by myoclonic and bilateral tonic-clonic seizures, who participated in a clinical protocol of ketogenic intermittent fasting, a method of intermittent fasting combined with medium-chain triglycerides (MCT) primarily designed for seizure management. The patient followed a 16:8 intermittent fasting regime combined with MCT intake for three months, followed by three months of all-alone intermittent fasting. Routine blood markers and brain MRI, including diffusion imaging were obtained before and after ketogenic fasting.
    Results: During the study protocol, while no seizure reduction in myoclonic seizures could be observed, ataxia - quantified by the Scale for the Assessment and Rating of Ataxia (SARA) - improved significantly from 8.5 to 6.0 during the interventions. MRI showed a trend suggesting improved cerebellar microstructural integrity.
    Conclusions: This case highlights the potential of ketogenic intermittent fasting as an adjunct therapy for mitochondrial ataxia. Ketogenic intermittent fasting was associated with clinically meaningful improvement of ataxia in a patient with COQ8A-related CoQ10 deficiency, suggesting that ketogenic dietary strategies may represent a promising adjunct therapeutic approach for mitochondrial ataxia. Future research should assess this intervention in larger patient cohorts to confirm its potential benefits.
    Keywords:  Ataxia; Coenzyme Q10 deficiency; IF-MCT study; Intermittent fasting; Ketogenic diet; Mitochondrial diseasse
    DOI:  https://doi.org/10.1016/j.ymgmr.2026.101296
  42. J Gerontol A Biol Sci Med Sci. 2026 Feb 17. pii: glag044. [Epub ahead of print]
      Aging significantly impacts brain function, and identifying reliable biomarkers for early detection of age-related neurodegeneration is crucial for improving diagnosis and treatment outcomes. This proof-of-principle study aims to evaluate the abundance of mitochondrial DNA (mtDNA) targets within plasma-derived extracellular vesicles (EVs) and to investigate whether they correlate with established biomarkers of brain aging, independent of chronological age and renal function. mtDNA copy number was quantified using absolute quantitative PCR (qPCR). Brain aging biomarkers were measured by ELISA. Multivariable regression analysis was performed to examine the associations between EVs mitochondrial genes and aging biomarkers. A multi-biomarker model was developed to assess the performance of combined biomarkers in distinguishing between age groups. We observed that EV mitochondrial gene levels were significantly increased with age (P < 0.001). Levels of neurofilament light chain (NfL), amyloid-beta (Aβ42 and Aβ40), also showed significant age-related increases (P < 0.001). A multi-biomarker model combining EVs mitochondrial genes and brain aging biomarkers showed the optimal performance in distinguishing older adults from younger individuals, with an area under the ROC curve (AUC) significantly higher than that of any single biomarker (P < 0.01). These findings collectively indicate that EV-derived mitochondrial genes, in combination with other biomarkers like NfL, hold great potential as a non-invasive tool for early detection and monitoring of brain aging and neurodegenerative diseases.
    Keywords:  Aging; Biomarker; Extracellular vesicles; Mitochondrial Genes
    DOI:  https://doi.org/10.1093/gerona/glag044
  43. iScience. 2026 Feb 20. 29(2): 114717
      Leigh syndrome is a fatal pediatric neurodegenerative disease caused by mitochondrial dysfunction, which can be modeled in the Ndufs4 KO mouse with mitochondrial respiratory chain complex I (CI) deficiency. This study explores NV354, a prodrug of succinate with enhanced oral bioavailability and brain uptake, as a potential therapy to counteract this devastating condition. NV354 modulated whole-body respiration and metabolic flexibility, prevented late-stage motor dysfunction, delayed clinical ataxia scores, and improved body weight development, but had otherwise minimal effect on neurobehavior and lifespan of the animals. The succinate prodrug prevented development of the brain stem lesions pathognomonic for Leigh syndrome, attenuated neuronal loss in the brainstem, diminished activation of astrocytes, blocked hypertrophic microglial accumulation, and reduced reactive oxygen species (ROS) levels in the brain. NV354 also partially alleviated motor symptoms and metabolic decompensation in a rat model of Parkinson disease induced by the CI inhibitor rotenone. In conclusion, the succinate prodrug NV354 shows promise as a potential treatment of mitochondrial CI-related neurodegeneration.
    Keywords:  biochemistry; biological sciences; natural sciences; neuroscience
    DOI:  https://doi.org/10.1016/j.isci.2026.114717
  44. Nat Commun. 2026 Feb 16.
      Pancreatic β-cells maintain glucose homeostasis by secreting insulin in response to rising blood glucose, a process fueled by mitochondrial ATP production. Iron, a core cofactor in the electron transport chain, is essential for this metabolic coupling. While the cytotoxic effects of iron overload are well known, the role of iron sufficiency during β-cell development remains unclear. Here, we identify a maturation-dependent requirement for iron in mouse and human β-cells. Using chemical chelation and genetic disruption of transferrin receptor (TFRC)-mediated uptake, we show that immature β-cells depend on iron during metabolic transition to functional maturity. Iron restriction at this stage impairs oxidative metabolism and compromises survival. In contrast, mature β-cells remain resilient to iron depletion, revealing a developmental switch in iron dependency. These findings establish iron as a key metabolic cue in β-cell development and suggest strategies to generate fully functional stem cell-derived β-cells for diabetes modeling and cell replacement therapy.
    DOI:  https://doi.org/10.1038/s41467-026-69574-y
  45. Commun Biol. 2026 Feb 14.
      Campylobacter jejuni, a major cause of bacterial gastroenteritis, is capable of surviving in diverse hosts, including free-living amoebae such as Acanthamoeba. However, the molecular mechanisms that facilitate its intracellular persistence and subsequent transfer remain poorly defined. Here, we hypothesize that C. jejuni employs a biphasic actin-remodelling strategy, mediated by the effector proteins CiaI and CiaD, to reposition and remodel host mitochondria, promoting mitochondrial aggregation and iron homoeostasis. Using dual proteomics, microscopy, biochemical assays, and defined genetic mutants, we show that actin polymerization and CiaI are critical for mitochondrial interaction. We found that CiaI binds nucleotides with cooperative kinetics, acting as a molecular switch, and is crucial for C. jejuni localization near mitochondria, while CiaD promotes actin polymerization and acanthopodia formation to facilitate uptake. We propose a two-phase model: early actin polymerization repositions mitochondria, followed by localized actin depolymerization and mitochondrial remodelling. Iron chelation promotes bacterial survival, suggesting that oxidative stress functions as a host defence. These findings highlight a sophisticated mechanism of intracellular adaptation by C. jejuni that may be relevant to pathogenesis and identify new potential targets for disrupting its environmental and clinical persistence.
    DOI:  https://doi.org/10.1038/s42003-026-09713-9
  46. Front Genet. 2026 ;17 1739328
       Background: Vanishing white matter disease (VWMD; OMIM 603896), also known as childhood ataxia with central nervous system hypomyelination (CACH), is a rare autosomal recessive leukodystrophy caused by pathogenic variants in the EIF2B gene family (EIF2B1-EIF2B5). Clinical manifestations are highly heterogeneous, with onset ranging from fetal life to adulthood; adult-onset cases remain relatively rare and often present with atypical symptoms. Brain magnetic resonance imaging (MRI) and genetic testing are pivotal for diagnosis.
    Case Presentation: We report a 32-year-old Chinese female with adult-onset VWMD characterized by intermittent headaches, progressive cognitive decline, menstrual irregularities, and hearing loss. Cranial MRI with diffusion-weighted imaging (DWI) revealed symmetrical periventricular and centrum semiovale white matter abnormalities. Whole-exome sequencing (WES) identified a homozygous missense variant in the EIF2B5 gene, formatted per Human Genome Variation Society (HGVS) guidelines as NM_001414.4:c.185A>T (p.Asp62Val). This variant was previously documented exclusively in a pediatric patient, representing the first report in an adult.
    Conclusion: Our case expands the phenotypic and age-related spectrum of EIF2B5-associated VWMD, highlighting that the c.185A>T variant is capable of manifesting in adulthood with non-classical features (e.g., headache as the initial symptom). Prior studies have confirmed that this variant impairs EIF2B complex function, which reinforces its pathogenic role in disrupting the integrated stress response (ISR) and maintaining white matter homeostasis. A literature review of 99 genetically confirmed adult-onset VWMD cases further underscores genotype-phenotype correlations: EIF2B5 is the most frequently mutated subunit in adult patients, with cerebellar ataxia, cognitive decline, and psychiatric symptoms as the predominant initial manifestations. Female patients often present with premature ovarian failure, a key diagnostic hallmark. Early genetic testing is crucial for definitive diagnosis, prenatal counseling, and symptomatic management. Notably, this study has limitations, including the lack of investigation into gene-gene interactions-factors that may modulate disease severity and phenotypic variability-and the unavailability of parental genetic data to fully validate zygosity.
    Keywords:  EIF2B5; adult-onset; genotype–phenotype correlation; homozygous variant; vanishing white matter disease
    DOI:  https://doi.org/10.3389/fgene.2026.1739328
  47. Nat Metab. 2026 Feb 20.
      Understanding protein distribution patterns across tissue architecture is crucial for deciphering organ function in health and disease. Here we show the application of single-cell Deep Visual Proteomics to perform spatially resolved proteome analysis of individual cells in native liver tissue. We built a robust framework comprising strategic cell selection and continuous protein gradient mapping, allowing the investigation of larger clinical cohorts. We generated a comprehensive spatial map of the human hepatic proteome by analysing hundreds of isolated hepatocytes from 18 individuals. Among the 2,500 proteins identified per cell, about half exhibited zonated expression patterns. Cross-species comparison with male mice revealed conserved metabolic functions and human-specific features of liver zonation. Analysis of samples with disrupted liver architecture demonstrated widespread loss of protein zonation, with pericentral proteins being particularly susceptible. Our study provides a comprehensive and open-access resource of human liver organization while establishing a broadly applicable framework for spatial proteomics analyses along tissue gradients.
    DOI:  https://doi.org/10.1038/s42255-026-01459-2
  48. Cell Death Dis. 2026 Feb 20.
      Human brain development is highly regulated by several spatiotemporal processes, which disruption can result in severe neurological disorders. Emerging evidence highlights the pivotal role of mitochondrial function as one of these fundamental pathways involved in neurodevelopment. Our study investigates the role of 4-hydroxyphenylpyruvate dioxygenase-like (HPDL) protein in cortical neurogenesis and mitochondrial activity, since mutations in the HPDL gene are associated with a childhood-onset form of hereditary spastic paraplegia characterized by corticospinal tract degeneration and cortical abnormalities. Starting from mutant neuroblastoma cells, we demonstrated that HPDL is important to respiratory chain supercomplex assembly and cellular redox balance. Moreover, RNA-seq studies revealed dysregulated pathways related to brain development. Generation of cortical neurons and organoids from HPDL patient-derived induced pluripotent stem cells exhibited premature neurogenesis at early differentiation stages, likely leading to depletion of cortical progenitors, as evidenced by decreased proliferation, slight increase of apoptosis, and unbalanced cortical type composition at later stages. Cortical organoids showed failure to grow at a normal rate, a feature highly reminiscent of the "microcephaly" observed in severe HPDL children. Mitochondrial morpho-functional characterization in mutant neurons confirmed disruption of OxPhos chain functionality in neuroblastoma knock-out model cells and HPDL mutant cortical progenitors also displayed defects in respirasome assembly and increased ROS generation rate. Treatment of mutant cortical cells with antioxidants and CoQ10 intermediates partially rescued premature neurogenesis depending on the mutational context, suggesting potential future personalized therapeutic strategies. Our findings reveal a critical role for HPDL in coordinating cortical progenitor proliferation, neurogenesis, and mitochondrial function, shedding light on a better understanding of the related clinical presentations.
    DOI:  https://doi.org/10.1038/s41419-026-08476-9
  49. iScience. 2026 Feb 20. 29(2): 114724
      Lipids are key structural and functional components of the brain, essential for cellular integrity and homeostasis. They regulate signaling pathways driving neuroinflammation, a key factor in many neurological disorders. We review the interplay between lipid metabolism and neuroinflammation, with a focus on very long-chain fatty acids, that are crucial for membrane integrity and of myelin formation. Insights from human genetic disorders highlight how lipid metabolic dysfunction triggers neuroinflammatory cascade. Studies in Drosophila now complement these findings by enabling rapid dissection of conserved pathways and testing of therapeutic strategies, including enzyme modulators, dietary interventions, and gene therapies. By bridging patient-derived insights with powerful in vivo modeling, Drosophila research is uncovering actionable mechanisms linking lipid dysregulation to neurodevelopmental and neurodegenerative disease.
    Keywords:  biochemistry; biological sciences; natural sciences
    DOI:  https://doi.org/10.1016/j.isci.2026.114724
  50. Lifestyle Genom. 2026 Feb 20. 1-10
       BACKGROUND: Creatine is a central regulator of cellular energy homeostasis and one of the most extensively studied dietary supplements in human nutrition. Although creatine supplementation consistently increases tissue creatine availability and supports performance and health across diverse populations, substantial interindividual variability in responsiveness persists. Approximately one-quarter of individuals demonstrate minimal increases in tissue creatine or functional benefit following supplementation. While non-genetic factors such as baseline creatine status, diet, age, sex, and training load contribute to this heterogeneity, the role of common genetic variation remains insufficiently explored. Importantly, creatine bioavailability and functional responsiveness are related but distinct outcomes, and both may be modified by genetic background.
    OBJECTIVE: This paper aims to reframe creatine responsiveness as a quantitative, polygenic trait shaped by common low-impact genetic variants rather than a binary responder-non-responder phenomenon driven by rare pathogenic mutations. The review synthesizes evidence on genetic variation affecting creatine transport, endogenous synthesis, and downstream energy metabolism, with an emphasis on population-relevant mechanisms.
    METHODS: A narrative, mechanism-oriented review was conducted integrating data from human genetics databases, biochemical pathways, and physiological studies. The analysis focused on (i) common low-impact variants in genes directly regulating creatine transport (SLC6A8) and biosynthesis (GATM, GAMT), and (ii) modifier genes involved in mitochondrial function, phosphagen buffering, and muscle or neural energetic phenotype. Variant classification frameworks from expert curation initiatives were used to distinguish pathogenic from low-impact population variants.
    RESULTS: Low-impact variants in the creatine transporter gene SLC6A8 are highly prevalent and likely contribute to a continuum of creatine transport efficiency, with sex-dependent effects due to X-linked inheritance. Similarly, common polymorphisms in creatine biosynthetic enzymes (GATM and GAMT) may subtly alter synthetic efficiency or methyl-group demand, increasing dietary creatine dependence while not causing overt deficiency. Beyond creatine-specific pathways, genetic variation in mitochondrial regulators, electron transport chain components, creatine kinase isoforms, and muscle fiber-type determinants can act as effect modifiers, amplifying or dampening the functional benefits of creatine despite comparable tissue uptake. Collectively, small additive effects across transport, synthesis, and utilization pathways may prevent supplementation from exceeding the threshold required for measurable benefit in certain individuals.
    CONCLUSIONS: Creatine non-responsiveness in the general population is more plausibly explained by the cumulative influence of common low-impact genetic variants than by rare monogenic defects. Viewing creatine responsiveness as a graded, polygenic trait provides a coherent framework to interpret heterogeneous findings in supplementation trials. Incorporating genetic context into study design, through stratified analyses or pathway-based approaches, may improve sensitivity to detect true effects and support the development of precision-guided creatine supplementation strategies in both clinical and public health settings.
    DOI:  https://doi.org/10.1159/000551090
  51. Sci Rep. 2026 Feb 20.
      Point mutations cause many genetic disorders, but modelling them in organisms is technically challenging. Creating mouse models that mimic these mutations is crucial for establishing a causal relationship between mutations and disease phenotype, thereby supporting the development of therapeutic strategies. Adenine base editors (ABEs) can correct single-nucleotide variants (SNVs) in disease modelling without double-stranded breaks (DSBs) or donor DNA, achieving higher product purity than traditional Cas9 methods. Earlier ABE techniques faced issues like limited targetability, bystander editing, and off-target effects. By combining two editor advancements, we introduced and tested ABE9-SpRY, an improved ABE variant fused with a PAM-flexible SpRY-Cas9 nickase. Our results show that ABE9-SpRY effectively generates three out of four targeted A-to-G mutations in mouse embryos, achieving desired editing efficiencies of up to 96% in individual adult founder mice. Furthermore, we observe fewer off-target events at predicted DNA sites in mouse embryos and in an orthogonal R-loop assay compared with ABE8e-SpRY. ABE9-SpRY also enhances product purity in mouse embryos under pooled sgRNA injections and, as a proof-of-concept, at a single endogenous locus in human induced pluripotent stem cells (hiPSCs), relative to ABE8e-SpRY. Our findings support ABE9-SpRY's precision at the loci tested and PAM-flexible versatility. Although performance remains sequence-dependent, these data support ABE9-SpRY as a PAM-flexible tool for generating precise point-mutation models where bystander editing is a concern.
    Keywords:  Adenine base editing; CRISPR-Cas9; Mouse modelling; Precision editing; hiPS cells
    DOI:  https://doi.org/10.1038/s41598-026-40642-z
  52. Nat Metab. 2026 Feb 18.
      Circadian clocks are internal timing systems that enable organisms to anticipate and adapt to daily environmental changes. These rhythms arise from a transcription-translation feedback loop in which CLOCK and BMAL1 regulate the expression of thousands of genes, including their repressors PER and CRY. Disruption of circadian rhythms contributes to obesity, metabolic disease and cancer, yet how the clock maintains metabolic homeostasis remains limited. Here we report that the clock regulates oxidative metabolism in adipocytes through diurnal complex I respiration. Disrupting the clock in male mice via adipocyte-specific genetic deletion or high-fat-diet feeding reduces complex I respiration in adipocytes, leading to suppression of the peroxisome proliferator-activated receptor and insulin signalling pathways. In contrast, restoring complex I function by expressing yeast NDI1 in adipocytes protects against diet-induced and circadian-induced metabolic dysfunction independently of weight gain. These findings reveal that adipocyte circadian disruption impairs metabolic health through mitochondrial complex I dysfunction, establishing clock control of complex I as a key regulator of metabolic homeostasis.
    DOI:  https://doi.org/10.1038/s42255-026-01464-5
  53. Cell Chem Biol. 2026 Feb 19. pii: S2451-9456(26)00031-0. [Epub ahead of print]33(2): 147-149
      The von Hippel-Lindau (VHL) protein is known for degrading hydroxylated proteins in normoxia, but its role under hypoxia remains unclear. In a recent report in Cell Metabolism Li et al.1 demonstrate that mitochondrial VHL remodels amino acid metabolism under chronic hypoxia to support cell growth, independent of hydroxylated protein degradation.
    DOI:  https://doi.org/10.1016/j.chembiol.2026.01.011
  54. Chem Res Toxicol. 2026 Feb 19.
      N-(1,3-Dimethylbutyl)-N'-phenyl-p-phenylenediamine Quinone (6PPDQ) not only causes acute mortality in salmon but also induces toxicities in other living organisms. The electrophilic quinone moiety in the 6PPDQ molecular structure can participate in binding to the cysteine residues that ubiquitously exist in protein nucleophiles, which are responsible for its toxicities. Out of the 82 6PPDQ-bonded proteins found in the human model cell line A549 on the sulfhydryl-reactive proteomics platform, which enables the precise identification of covalent binding protein targets of a pollutant, we discovered three 6PPDQ-bonded mitochondrial proteins─NDUS6, COX5B, and ATP5PB─that are involved in mitochondrial dysfunction for the first time. They impede the function of mitochondria, as witnessed by the decreased enzymatic activities of mitochondrial respiratory chain Complexes I (27.63%) and IV (23.11%), the decreased cellular ATP content (19.94%), and the reduced mitochondrial membrane potential (3.2-fold), as well as the elevated mitochondrial ROS level (2.2-fold) under the environmentally relevant 8.9 μg/L 6PPDQ exposure compared to the controls. Our findings provide experimental evidence for elucidating 6PPDQ's toxicities at the molecular level, and the knowledge learned will enhance the public's awareness of the adverse impacts of environmental pollution on health.
    DOI:  https://doi.org/10.1021/acs.chemrestox.5c00250
  55. Nature. 2026 Feb 18.
      
    Keywords:  Gene therapy; Medical research; Neuroscience
    DOI:  https://doi.org/10.1038/d41586-026-00291-8
  56. NPJ Genom Med. 2026 Feb 14.
      Digital platforms hold promise to scale implementation of population screening. We tailored the Genetics Adviser platform to provide education, decision support, consent, and result return in a genomic newborn screening (gNBS) study, BabyScreen + . Participants were surveyed and interviewed on the usability and value of Genetics Adviser. Genetics Adviser was used by 1048 participants and 1007 (96%) provided feedback. The majority (96%, n = 963) found the platform easy to navigate, with 85% (n = 851) spending <20 min online. Participants demonstrated excellent understanding, over 80% answering at least 6/8 knowledge questions correctly. Only 7% (12/173) of participant-initiated contacts with the study team were for genetic counselling. Interview participants valued the online process. We demonstrate the successful use of a digital platform for a genomic screening program. This model is streamlined, providing consistent, user-friendly education to support decision-making with minimal input from healthcare practitioners. Further evaluation in diverse populations will be essential for future use.
    DOI:  https://doi.org/10.1038/s41525-026-00551-6
  57. J Inherit Metab Dis. 2026 Mar;49(2): e70143
      A critical concern of newborn screening (NBS) for very-long chain acyl-CoA dehydrogenase deficiency (VLCADD) is the difficulty of predicting clinical outcomes. To address this, we investigated neonatal C18:2-carnitine concentrations as a possible predictor of VLCADD phenotype. To investigate the impact of sex, gestational age (GA) at birth, sampling day and birth weight on C18:2-carnitine, we analyzed NBS-dried blood spots (DBS) from Dutch newborns born between 2018 and 2020 (n = 209.785). After normalization for resulting confounders, C18:2-carnitine concentrations were investigated in NBS-DBS (n = 15) and neonatal plasma (n = 35) of Dutch VLCADD-patients, and in German NBS-DBS (n = 6) and correlated with clinical severity and diagnostic assays. Results showed that C18:2-carnitine concentrations were affected by GA, sampling day, birth weight and, to a lesser extent, by sex. High C18:2-carnitine, normalized for GA, sampling day and birth weight, reliably identified all VLCADD-patients with (expected) severe phenotypes. The differentiating C18:2-carnitine was identified as linoleylcarnitine. In conclusion, this study shows that neonatal C18:2-carnitine concentrations can serve to predict disease severity directly after positive NBS for VLCADD. Patients with high C18:2-carnitine concentrations can be considered "severe" and require strict dietary treatment and close monitoring. Patients with low C18:2-carnitine concentrations can be identified as "mild" and only need preventive dietary measures.
    Keywords:  C18:2‐carnitine; newborn screening; prognosis prediction; very long‐chain acyl‐CoA dehydrogenase deficiency (VLCADD)
    DOI:  https://doi.org/10.1002/jimd.70143
  58. Biochim Biophys Acta Mol Basis Dis. 2026 Feb 15. pii: S0925-4439(26)00043-8. [Epub ahead of print]1872(4): 168195
      Aberrant endoplasmic reticulum (ER) and mitochondria function mediated by deregulated levels of tethering proteins at the mitochondria-associated ER membranes (MAM) sites is a hallmark of several diseases, yet very little is known of the regulatory mechanisms of these tethering proteins. Here, using mouse hepatic cells, we present data to show that miR-3099-5p binds to the 3'UTR of one such MAM protein, FACL4 and regulates its levels within the cell. Hepatic levels of miR-3099-5p are up-regulated during diabetes with a concomitant down-regulation of FACL4 levels. Overexpressing miR-3099-5p levels in mouse Hepa1-6 cells effectively down-regulates FACL4 levels, the effect being prevented by the miR-3099-5p inhibitor. miR-3099-5p-FACL4 interaction leads to accumulation of arachidonic acid and its reduced incorporation into phospholipids. Further, while this interaction did not impact mitochondrial ROS or calcium levels, it altered mitochondrial membrane potential and mitochondrial permeability transition pore opening together with increased apoptosis. FACL4 inhibition alone exerted effects similar to miR-3099-5p overexpression including accumulation of arachidonic acid, altering mitochondrial membrane potential and mitochondrial permeability transition pore opening and induction of apoptosis. Interestingly, arachidonic acid overloading in Hepa1-6 cells was sufficient to induce apoptosis and impair mitochondrial membrane potential; such arachidonic acid supplementation also significantly reversed the effects of miR-3099-5p inhibition on apoptosis and mitochondrial membrane potential. These findings suggest that FACL4 mediates the deleterious effects of miR-3099-5p in mouse hepatic cells and interrogating such miRNA mediated changes in hepatic FACL4 levels might be explored to address aberrant hepatic metabolism during diabetes.
    Keywords:  Apoptosis; ER-mitochondria function; FACL4; Mitochondrial dysfunction; miRNA
    DOI:  https://doi.org/10.1016/j.bbadis.2026.168195
  59. FASEB J. 2026 Feb 28. 40(4): e71582
      Nicotinamide adenine dinucleotide (NAD+) is a vital molecule, serving as a redox cofactor and the limiting substrate for numerous enzymes. NAD+ decline is a key feature of aging, while supplementation with NAD+ precursors can efficiently counteract aging traits and prevent age-associated conditions in preclinical models. However, clinical translation remains challenging, likely due to the limited NAD+ boosting capacity of classical precursors, such as nicotinamide mononucleotide (NMN) and nicotinamide riboside (NR). This has brought attention to their reduced forms, reduced NMN (NMNH) and reduced NR (NRH), which are more potent NAD+ boosters but remain poorly characterized. Here, we performed a comprehensive comparative analysis using RNA sequencing, proteomics, and metabolomics on cultured murine hepatocytes treated with NMN, NMNH, NR, or NRH. Global metabolic profiling revealed that NRH and NMNH induced substantially broader metabolic alterations than NR and NMN, with NRH uniquely suppressing metabolites involved in energy metabolism. The pronounced metabolic effects were reflected at a transcriptional level, with reduced precursors triggering a significantly higher number of differentially expressed genes than oxidized ones. Shared differentially expressed genes between NMNH and NRH revealed upregulation of stress-related glutathione-S-transferases (Gsts) which furthermore were reflected in our proteomic profiling. However, the upregulation of Gsts did not cause a depletion of glutathione or oxiglutathione, suggesting a pseudo-stress response to reduced NAD+ precursors. Together, our data demonstrate that reduced NAD+ precursors are unique and distinct from the market-available NAD+ precursors NR and NMN, not only as more potent NAD+ boosters, but also as compounds influencing a broader range of cellular processes.
    Keywords:  NAD+; NAD+ precursors; comparative analysis; hepatocytes
    DOI:  https://doi.org/10.1096/fj.202501925R
  60. Cell Metab. 2026 Feb 17. pii: S1550-4131(26)00012-4. [Epub ahead of print]
      Endothelial cells (ECs) are key metabolic gatekeepers, yet their role in metabolic health remains unclear. Given their central involvement in energy metabolism, mitochondria are ideally positioned to enable ECs to adapt to ever-changing metabolic requirements. Here, we explore the hypothesis that mitochondrial dynamics proteins in ECs influence whole-body metabolic status. Genetic deficiency of Mfn2 in ECs (Mfn2iΔEC), but not of Mfn1iΔEC, induces a mitohormetic response in the adipose vasculature, enhancing antioxidant defenses, mitochondrial fitness, and lipid oxidation, ultimately improving metabolic outcomes. Cultured ECs secrete the mitokine growth differentiation factor 15 (GDF15) via a forkhead box O1 (FOXO1)-dependent axis, a response also observed under stress conditions in vivo. Notably, Mfn2iΔEC mice exhibited elevated endothelial and circulating GDF15 levels, and neutralization of GDF15 partly attenuated their metabolic benefits. Consistent with mitohormetic activation, Mfn2iΔEC mice showed protection against diet-induced obesity and delayed age-related decline. Hence, vascular mitohormetic adaptations emerge as a novel mechanism promoting systemic metabolic health.
    Keywords:  GDF15; aging; diabetes; endothelial cells; mitochondria; mitofusin; mitohormesis; obesity
    DOI:  https://doi.org/10.1016/j.cmet.2026.01.012
  61. Cell Rep Methods. 2026 Feb 17. pii: S2667-2375(26)00031-7. [Epub ahead of print] 101331
      Cancer cachexia, a devastating metabolic wasting syndrome affecting up to 80% of solid cancer patients, remains incurable despite advances in tumor biology understanding. This study introduces neuromuscular organoids (NMOs) derived from human-induced pluripotent stem cells (hiPSCs) as a platform to investigate cancer-driven muscle cachexia. We found that NMOs respond well to atrophic stimuli and replicate the key features of cancer cachexia when treated with conditioned media derived from cachexia-inducing cancer cells. Specifically, cachectic NMOs showed muscle mass loss, impairment of muscle contraction, alteration of intracellular calcium homeostasis, appearance of mitochondrial dysfunction with a metabolic shift, and enhancement of autophagy. Based on these results, we propose NMOs derived from hiPSCs as an in vitro tool for investigating human muscle cachexia, with potential future avenues of patient-specific modeling and therapeutic screening.
    Keywords:  CP: cancer biology; CP: stem cell; autophagy; cancer cachexia; human induced pluripotent stem cells; in vitro human disease model; metabolic remodeling; mitochondrial dysfunction; neuromuscular junction; neuromuscular organoid; skeletal muscle wasting
    DOI:  https://doi.org/10.1016/j.crmeth.2026.101331
  62. Biomaterials. 2026 Feb 11. pii: S0142-9612(26)00095-5. [Epub ahead of print]330 124071
      Mitochondria are essential for cell energy metabolism, redox homeostasis, and apoptosis. Meanwhile, numerous pathological conditions are linked with mitochondrial dysfunction, such as cardiovascular diseases, connective tissue disorders, chronic wounds, neurological disorders, and cancer. Mitochondria-targeted hydrogels (MTHs) have emerged for their ability to selectively deliver active agents to mitochondria, modulate mitochondrial function precisely, which plays a key role in improving treatment efficacy. Hydrogels offer unique advantages, including biocompatibility, structural tunability, and controlled drug release. With specific targeting ligands and stimuli-responsive mechanisms, hydrogels can achieve mitochondrial localization and therapeutic modulation. Recent advancements have demonstrated significant benefits of MTHs in reducing oxidative stress, promoting oxidative phosphorylation and restoring mitochondrial quality control across a variety of disease models. However, challenges remain, including optimizing targeting efficiency, as well as accuracy. Further exploration of therapeutic mechanisms and the integration of multi-dimensional targeting strategies are also essential for the clinical application of MTHs. The current review highlights the development of mitochondrial targeting strategies and specifically focuses on a series of applications of MTHs in mitochondrial-related diseases. Lastly, the discussion delves into the shortcomings of existing therapies and possible future research ideas.
    Keywords:  Hydrogel engineering; Mitochondria-related diseases; Mitochondria-targeted hydrogels; Mitochondrial quality control; Tissue regeneration
    DOI:  https://doi.org/10.1016/j.biomaterials.2026.124071
  63. Nat Commun. 2026 Feb 14. 17(1): 1698
    Solve-RD consortium
      Most patients with a rare movement disorder (MD) do not receive a molecular diagnosis, and the underlying genetic variants and mediating genes remain elusive. Here, we evaluate the diagnostic accuracy of conventional and next-generation sequencing-based genetic testing strategies in a cohort of 2,811 individuals with ataxia, spastic paraplegia and dystonia. Exome sequencing establishes genetic diagnoses in 19.3% of cases, and specificity of phenotypic features and age at testing are positive predictors. Genome analysis 'beyond the exome' increases the diagnostic yield by 7.5%, mostly due to the improved detection of structural variants and repeat expansions. Unsolved cases are included in the Solve-RD cohort and subjected to gene-burden analysis, providing evidence for loss-of-function variants in X-chromosomal CD99L2 causing spastic ataxia. Cellular studies show that the transmembrane protein CD99L2 occurs mainly in a ubiquitinated form and serves as an activating interactor of the calcium-dependent protease CAPN1. Ablation of cytoplasmic or extracellular domains of CD99L2 leads to its intracellular mislocalization and abrogation of its interplay with CAPN1. Transcriptome analysis in CD99L2 patient-derived fibroblasts reveals synaptic function-specific disturbances. Impaired CAPN1 activation and dysregulation of downstream neuronal pathways constitute the likely molecular cause for neurodegeneration.
    DOI:  https://doi.org/10.1038/s41467-026-69337-9
  64. Nat Commun. 2026 Feb 14.
      Conventional storage and retrieval of nucleic acid specimens, particularly unstable RNA, rely on costly cold-chain infrastructure and inefficient robotic handling, inhibiting large-scale nucleic acid archives needed for global genomic biobanking. We introduce a scalable room-temperature storage system with minimal physical footprint that enables database-like queries on encapsulated, barcoded, and pooled nucleic acid samples. Queries incorporate numerical ranges, categorical filters, and combinations thereof, advancing beyond previous demonstrations of single-sample retrieval or Boolean classifiers. We evaluate this system on ninety-six mock SARS-CoV-2 genomic samples barcoded with theoretical patient data including age, location, and diagnostic state, demonstrating rapid, scalable retrieval. We further demonstrate storage and sequencing of human patient-derived nucleic acid samples, illustrating applicability to clinical genomic analysis. By avoiding freezer-based storage and retrieval, this approach scales to millions of samples without loss of fidelity or throughput, enabling large-scale pathogen and genomic repositories in under-resourced or isolated regions of the US and worldwide.
    DOI:  https://doi.org/10.1038/s41467-026-69402-3
  65. J Nutr. 2026 Feb 18. pii: S0022-3166(26)00076-3. [Epub ahead of print] 101427
       BACKGROUND: Mitochondrial dysfunction is recognized as a key pathophysiological mechanism in neurodegenerative diseases. Alterations in mitochondrial dynamics-including imbalances in fission and fusion, impaired biogenesis, and disrupted mitophagy-contribute to the onset and progression of neurological disorders. In this context, mitochondrial modulation has emerged as a promising therapeutic strategy.
    OBJECTIVE: This systematic review examined the role of riboflavin, a water-soluble vitamin and essential mitochondrial cofactor, in neurological interventions through mitochondrial modulation, with emphasis on elucidating the underlying molecular mechanisms.
    METHODS: A search of the PubMed, Embase, Scopus, and Web of Science databases identified 23 eligible studies, comprising 6 in vitro experiments, 10 rodent models, and 7 clinical trials.
    RESULTS: These studies evaluated the effects of riboflavin in monogenic, neurodegenerative, and demyelinating mitochondrial diseases, cerebrovascular/hypoxic injury, and pain/migraine. Clinical evidence indicated that riboflavin may regulate oxidative stress in stroke and perinatal asphyxia, with associated functional improvements. Preclinical findings revealed mechanisms of action involving energy homeostasis, cell cycle regulation, and mitochondrial dynamics across monogenic mitochondrial disorders, neurodegenerative diseases, hypoxic injury, and models of pain and migraine. Possibly through mitochondrial modulation, riboflavin appeared to reduce α-synuclein aggregation in Parkinson's disease, increase the number of tyrosine-hydroxylase-positive neurons in Alzheimer's disease models, enhance neuronal survival in Brown-Vialetto-Van Laere and Huntington's disease models, and normalize neuronal excitability in ataxia and migraine. In contrast, no therapeutic effects were observed in demyelinating diseases.
    CONCLUSIONS: Overall, the findings suggest that riboflavin may promote neuroprotection through redox modulation and gene regulation, stabilization of membrane potential, and enhanced mitochondrial complex activity via flavin cofactors, ultimately supporting neuronal metabolism and functional outcomes. Despite advances in mechanistic understanding, clinical applications in humans remain insufficiently defined for most conditions, with clearer dosage regimens currently established only for stroke and migraine.
    Keywords:  brain; degenerative diseases; mitochondria; mitochondrial biogenesis; vitamin B2
    DOI:  https://doi.org/10.1016/j.tjnut.2026.101427