bims-proteo Biomed News
on Proteostasis
Issue of 2026–09–20
fifty-nine papers selected by
Eric Chevet, INSERM



  1. Mol Cell. 2026 Sep 17. pii: S1097-2765(26)00585-X. [Epub ahead of print]86(18): 3803-3821.e17
      Rapid protein turnover is essential for cellular stress adaptation. HUWE1 (HECT, UBA, and WWE domain containing 1), a large HECT-type E3 ligase, regulates many short-lived stress-responsive proteins, yet the mechanisms underlying its substrate selectivity remain unclear. Here, we reveal that HUWE1 functions as a ubiquitin chain amplifier that captures pre-ubiquitinated substrates and amplifies the degradation signal by assembling long ubiquitin chains containing K11-K48 branch points, a process regulated by its partners HUWE1-associated protein stress response 1 (HAPSTR1) and USP7 (ubiquitin-specific-processing protease 7). Structural and biochemical analyses show that HAPSTR1 engages HUWE1's ubiquitin-binding motifs to drive nuclear import and modulate substrate recruitment. A cryo-EM structure of the HUWE1-USP7 complex reveals a bidirectional regulatory mechanism: HUWE1 activates USP7's catalytic activity, while USP7 modulates HUWE1 conformational states. Global proteomic analyses demonstrate that this axis drives extensive remodeling of the short-lived nuclear proteome. These findings establish the HUWE1-HAPSTR1-USP7 complex as a key ubiquitin code modifier, providing a molecular rationale for HUWE1 dysregulation in neurodevelopmental disorders and cancer.
    Keywords:  HAPSTR1; HECT ubiquitin ligase; HUWE1; UPS; USP7; branched ubiquitin chains; cryo-EM; nuclear proteostasis; p53; ubiquitin chain amplification
    DOI:  https://doi.org/10.1016/j.molcel.2026.08.016
  2. Matrix Biol. 2026 Sep 16. pii: S0945-053X(26)00094-6. [Epub ahead of print] 102052
      Collagens are synthesized in the endoplasmic reticulum (ER), where their biosynthesis, quality control, and secretion are tightly coordinated to maintain collagen proteostasis. TANGO1L is an ER exit site protein required for efficient collagen secretion; however, whether it contributes equally to the secretion and intracellular homeostasis of different collagen types remains unclear. Here, we systematically examined the consequences of TANGO1L deficiency using CRISPR/Cas9-generated TANGO1L knockout human fibroblasts. Loss of TANGO1L reduced the secretion of multiple major collagen types and delayed secretion of newly synthesized collagen I. Intracellular responses, however, differed markedly among collagen types: collagen V accumulated intracellularly, whereas collagens I, III, and IV avoided intracellular retention despite markedly reduced extracellular secretion. This differential response was accompanied by decreased collagen gene expression and selective remodeling of the ER collagen biosynthetic machinery, suggesting that these cells adjust collagen synthesis to a reduced secretory capacity. TANGO1S protein was consistently upregulated through a post-transcriptional mechanism, suggesting an adaptive response to chronic TANGO1L deficiency. Biochemical analysis of collagen purified from the culture medium revealed a trend toward modestly increased lysine post-translational modifications, including hydroxylysine glycosylation, without altered triple-helical structure or thermal stability, indicating that TANGO1L deficiency compromises the quantity rather than the quality of secreted collagen. Together, these findings establish that the requirement for TANGO1L differs among collagen types and that its loss induces adaptive remodeling of intracellular collagen proteostasis rather than simply impairing collagen secretion, providing a framework for understanding how secretory cells maintain collagen homeostasis when secretion is compromised.
    Keywords:  ER exit sites; TANGO1; collagen; proteostasis; secretion
    DOI:  https://doi.org/10.1016/j.matbio.2026.102052
  3. Sci Adv. 2026 Sep 18. 12(38): eaeh8258
      Signal recognition particle (SRP), a universally conserved protein-targeting machine, cotranslationally recognizes signal sequences of nascent endomembrane proteins and engages the SRP receptor (SR) at the endoplasmic reticulum (ER) membrane to unload the translating ribosome. The mechanism of membrane-associated steps in this pathway remains unclear. Here, we address this question using pre-steady-state and single-molecule fluorescence measurements. SR induces release of the signal sequence from SRP and detachment of the SRP guanosine triphosphatase (GTPase) domain from the ribosome. Despite the dissolution of these contacts, SR acts as a tether to stabilize SRP association with the ribosome. Guanosine 5'-triphosphate (GTP) hydrolysis in the SRP-SR complex abolishes this stabilizing effect to facilitate ribosome-nascent chain complex (RNC) release from SRP. The ER membrane selectively promotes a prehandover conformation of the RNC-SRP-SR complex, in which delayed GTP hydrolysis optimizes the transition from targeting to translocation. Our findings provide a high-resolution model for key steps during cargo handover at the ER and highlight the critical role of fine-tuned GTPase activity in the SRP pathway.
    DOI:  https://doi.org/10.1126/sciadv.aeh8258
  4. Nat Cell Biol. 2026 Sep 15.
      Lysosomal membrane integrity is essential for preserving cellular homeostasis in response to different stressors. Upon lysosomal membrane permeabilization, cells activate several mechanisms for lysosomal membrane repair, including ESCRT proteins, phosphatidylinositol 4-phosphate (PI4P)-dependent lipid transfer from the endoplasmic reticulum (ER) and conjugation of ATG8 family proteins to single membranes (CASM). The interplay between these pathways and the regulation of the lipid transfer machinery remain incompletely understood. Here we show that phosphatidylinositol 3-phosphate (PI3P)-containing ER domains play a major role in lysosomal membrane repair. PI3P is formed on lysosome-proximal ER domains by the phosphatidylinositol 3-kinase PIK3C3/VPS34 in response to membrane damage, and inhibition or depletion of PIK3C3 inhibits lysosome repair. Mechanistically, the ATPase DFCP1/ZFYVE1 accumulates on lysosome-proximal ER domains by its PI3P binding, triggered by Ca2+ efflux from lysosomes and requiring the ULK1 kinase complex and ER proteins of the VAP family. Downstream of CASM, PI4P, ESCRTs and PI3P, DFCP1 promotes focal accumulation of the lipid channel VPS13C on ER domains proximal to damaged lysosomes to promote their repair. The function and dynamics of DFCP1 depend on its ability to bind and hydrolyse ATP, and absence of DFCP1 compromises cellular resistance to vacuolar damage induced by Listeria monocytogenes. We conclude that DFCP1 mediates concentration of the ER-associated lipid transport machinery at damaged lysosomes to promote their sealing in response to Ca2+ flux and PIK3C3 activation.
    DOI:  https://doi.org/10.1038/s41556-026-02062-z
  5. Nature. 2026 Sep 15.
      Quality control of biomolecules is vital for organismal health. While DNA repair and protein quality control are well understood, how cells monitor other important biomolecules such as glycogen remains ill-defined. The accumulation of aberrant, poorly branched glycogen into insoluble polyglucosan bodies causes severe disease1,2. Here, we discover autophagy of ubiquitylated aberrant glycogen as a previously unrecognized quality control mechanism safeguarding the brain from polyglucosan buildup. This mechanism depends on the E3 ubiquitin ligase RNF213. Mice lacking ligase activity in RNF213 accumulate polyglucosan in cerebellum, pons, and hippocampus. Using cells engineered to produce polyglucosan, we show that RNF213 selectively ubiquitylates abnormal glycogen. Cryo-EM analysis of RNF213 bound to glycogen-derived maltoheptaose revealed its CBM20 domain binds linear oligosaccharides. Disrupting carbohydrate binding results in gain of E3 ligase activity towards physiological glycogen, indicating the CBM20 domain limits RNF213 activity towards physiological glycogen. Epistasis analysis places RNF213 upstream of LUBAC, suggesting a hierarchical network of multiple E3 ligases surveying glycogen quality. Ubiquitylated polyglucosan recruits the autophagy receptors SQSTM1, TAX1BP1, and optineurin, thereby triggering uptake into autophagosomes. These findings identify RNF213 as a quality control factor preventing polyglucosan accumulation in astrocytes through direct ubiquitylation of polyglucosan, revealing an essential role for non-protein ubiquitylation in glycogen quality control.
    DOI:  https://doi.org/10.1038/s41586-026-11139-6
  6. Protein Sci. 2026 10;35(10): e70780
      In numerous neurodegenerative diseases known collectively as tauopathies, the microtubule-associated protein tau forms fibrillar aggregates that are hallmarks of disease pathology. Tauopathies represent a substantial fraction of diseases associated with protein misfolding. Cellular chaperones known as small heat shock proteins (sHSPs) play a critical role in maintaining protein homeostasis by delaying the onset of protein aggregation. Two sHSPs, HSPB1 (Hsp27) and HSPB5 (αB-crystallin), are constitutively expressed in the brain and neurons. Here, we show that HSPB1 and HSPB5 delay tau aggregation in vitro through distinct mechanisms dictated by their disordered N-terminal regions (NTRs). HSPB1 inhibits tau aggregation under normal cellular conditions, whereas HSPB5 displays activity toward tau when activated by stress conditions such as pH acidosis. Using chimeric HSPB1/HSPB5 constructs in which small NTR subregions are swapped, we identify functional regions within the NTRs that modulate chaperone function for tau. The functional regions contain known sites of phosphorylation, suggesting that they are also control points that respond to cellular stress conditions. Our findings support an emerging model in which specific functional motifs within disordered regions of sHSPs govern activity and client engagement under normal and stress conditions.
    Keywords:  HSPB1; HSPB5; Hsp27; chaperone; intrinsic disorder; protein aggregation; small heat shock proteins; tau aggregation; αB‐crystallin
    DOI:  https://doi.org/10.1002/pro.70780
  7. Proc Natl Acad Sci U S A. 2026 Sep 22. 123(38): e2616684123
      Lysosomal enzymes are synthesized in the Endoplasmic Reticulum (ER) and transported to lysosomes to execute their functions. Deficiencies in lysosomal enzymes or components of the lysosomal transport machinery result in lysosomal storage disorders. While mannose-6-phosphate mediated lysosomal enzymes sorting in the Golgi has been extensively characterized, the mechanisms governing their export from the ER remain elusive. Here, we show that de novo lipogenesis, a metabolic pathway responsible for fatty acid synthesis, regulates lysosomal enzyme transport. Inhibition of de novo lipogenesis leads to the retention of lysosomal enzymes within the ER. Mechanistically, fatty acid derived from de novo lipogenesis is used for Arf1 myristoylation. Myristoylated Arf1 promotes retrograde vesicle trafficking from the Golgi to the ER, thereby maintaining the homeostatic bidirectional flux required for efficient ER export of lysosomal enzymes. Our findings uncover a critical functional link between lipid metabolism and lysosomal enzyme trafficking.
    Keywords:  SREBP; de novo lipogenesis; lysosomal enzyme transport; protein myristoylation; proximity labeling
    DOI:  https://doi.org/10.1073/pnas.2616684123
  8. Biochem Soc Trans. 2026 Sep 23. 54(9): 1251-1260
      TRIpartite Motif (TRIM) family proteins are required for healthy development and homeostasis, while their dysregulation is observed in a wide range of diseases. Although diverse cellular roles are emerging for TRIMs, the majority function as ubiquitin E3 ligases by means of their RING (really interesting new gene) domain. Here, we delve into what is known about TRIM ubiquitin E3 ligase mechanisms, their ubiquitin chain specificities, and how they can be regulated through homo- and hetero-multimerisation and post-translational modifications.
    Keywords:  E3 ligase; TRIM; Ubiquitin; catalytic mechanisms; structural biology; targeted protein degradation
    DOI:  https://doi.org/10.1042/BST20260079
  9. Nat Commun. 2026 09 16. pii: 9877. [Epub ahead of print]17(1):
      Cellular senescence is a state of irreversible cell cycle arrest triggered by telomere erosion, persistent DNA damage or chronic stress. The accumulation of senescent cells disrupts tissue function and contributes to aging and disease. Here, we employ mass spectrometry-based proteomics to systematically interrogate dynamic proteome changes at multiple levels during the progression of replicative cellular senescence. We demonstrate that proteome changes during senescence occur in a coordinated manner, characterized by widespread protein depletion on chromatin. Moreover, components of the cytoplasmic translation machinery are depleted, while mitochondrial proteins display increased insolubility. Autophagic and proteasome activity is compromised in senescent cells along with remodeling of ubiquitin linkages and depletion of ubiquitin E3 ligases. Comparison of the senescent proteome with different pathophysiological cellular states reveals a distinctive senescent signature shaped by changes in the proteostasis network. Collectively, we provide a resource for the exploration of temporally resolved changes in the senescent proteome.
    DOI:  https://doi.org/10.1038/s41467-026-77686-8
  10. Nat Commun. 2026 09 16. pii: 9647. [Epub ahead of print]17(1):
      Efficient clearance and recycling of dysfunctional mitochondria through the robust catabolic activity of lysosomes are essential for cellular health. However, how membrane lipids contribute to maintaining the degradative capacity of lysosomes remains poorly understood. Here, we show that cholesterol plays a critical role in preserving the functional integrity of degradative lysosomes. Clearance of damaged mitochondria by degradative lysosomes is tightly coupled with the acute accumulation of phosphatidylinositol 4-phosphate (PI4P) on the lysosomal surface via PI4KIIα activity. This PI4P accumulation activates oxysterol-binding protein (OSBP)-mediated cholesterol transport from the endoplasmic reticulum (ER) to lysosomal membranes. The resulting efflux of cholesterol from the ER activates sterol regulatory element-binding protein 2 (SREBP-2), enhancing cholesterol production. Sustained cholesterol accumulation on lysosomal membranes maintains lysosomal acidity and membrane integrity for efficient mitochondrial degradation. This degradation process then leads to the release of free fatty acids and their recycling and storage through the formation of DGAT1-dependent lipid droplets. These findings uncover a key phosphoinositide-regulated cholesterol transport pathway that promotes the clearance and recycling of dysfunctional mitochondria, a process whose impairment is closely linked to neurodegeneration.
    DOI:  https://doi.org/10.1038/s41467-026-77423-1
  11. Cell. 2026 Sep 14. pii: S0092-8674(26)01004-4. [Epub ahead of print]
      Programmed ribosomal frameshifting (PRF) is a conserved viral strategy for expressing polyproteins from compact genomes. Although PRF is traditionally viewed as a structural mechanism, here we show that it functions as a regulatory signal that rewires host translation in favor of viral replication. A minimal SARS-CoV-2 PRF element is sufficient to activate the GCN2 arm of the integrated stress response (ISR) independently of the canonical ISR sensor ZAKα. This activation serves as a temporal switch during early infection to shut off host translation and is required for viral propagation in cells and human airway organoids. Proteomic and genetic screens identify DRG1 and IGF2BP3 as key mediators of PRF-induced GCN2 activation. We further show that this PRF-GCN2 axis is conserved in human immunodeficiency virus (HIV)-1 and West Nile virus, highlighting its broad relevance across RNA viruses. These findings reveal a sophisticated mechanism of viral translational control, highlighting PRF as a stress-inducing module that enhances viral replication.
    Keywords:  RNA virus; cellular stress; host-pathogen interactions; integrated stress response; programmed ribosomal frameshifting; ribosome collision; translation; virology
    DOI:  https://doi.org/10.1016/j.cell.2026.08.031
  12. Nat Commun. 2026 Sep 17. pii: 9882. [Epub ahead of print]17(1):
      The tRNA ligase complex (tRNA-LC) seals tRNA exon halves in the nucleus during pre-tRNA splicing and XBP1-mRNA exons in the cytoplasm as part of the unfolded protein response (UPR). This dual function requires the tRNA-LC to be either nuclear or cytoplasmic. Here, we reveal that Ashwin (ASW), the vertebrate-specific subunit of the tRNA-LC, serves as its nuclear import factor. ASW contains a dual nuclear localisation signal (NLS) which, upon disruption, leads to the retention of the tRNA-LC in the cytoplasm, impairing pre-tRNA splicing with the consequent accumulation of 5' tRNA fragments. We also show that the tRNA-LC exists in three forms, depending on which FAM98 paralog is bound, either FAM98A, FAM98B or FAM98C. ASW interacts exclusively with the FAM98B-containing complex, ensuring its nuclear localization for tRNA biogenesis. Attaching an NLS to RTCB, the catalytic and indispensable tRNA-LC subunit, rescues pre-tRNA splicing in cells depleted of ASW. We hypothesize that vertebrates evolved ASW to localize a sub-population of tRNA-LC to the nucleus, while using FAM98 paralogs to retain a fraction of RTCB in the cytoplasm to splice XBP1-mRNA during UPR.
    DOI:  https://doi.org/10.1038/s41467-026-77451-x
  13. Mol Cell Proteomics. 2026 Sep 18. pii: S1535-9476(26)00159-3. [Epub ahead of print] 101663
      Phosphodiesterase 3A (PDE3A) modulators such as anagrelide induce complex formation between PDE3A and Schlafen 12 (SLFN12), selectively killing cancer cells that co-express both proteins. As PDE3A forms isoform- and cell type-specific signalosome complexes across multiple subcellular compartments, the effects of its modulation are expected to depend strongly on the surrounding protein interaction network. However, despite considerable pre-clinical and early clinical interest in PDE3A modulators, the cellular context in which these compounds act has remained poorly characterized. Using proximity-dependent biotinylation in two human cancer cell lines (SA-4, liposarcoma; HeLa, cervical adenocarcinoma), we mapped the interactomes of PDE3A, SLFN12, and the anagrelide-induced PDE3A-SLFN12 complex. Anagrelide induced 259 high-confidence interactions, most notably with ribosomal proteins and translation initiation factors, while suppressing 877 interactions, most prominently those associated with the proteasome, protein folding, and the ER membrane. Interactions were selectively induced with peripheral eukaryotic translation initiation factor 3 (eIF3) subunits eIF3A and eIF3B, while interactions with core eIF3 subunits eIF3H, eIF3L, and eIF3M were suppressed. These findings were validated by native co-immunoprecipitation across three cancer cell lines, and multiplex immunofluorescence confirmed accumulation and ribosomal redistribution of both PDE3A and SLFN12 following anagrelide treatment. AlphaFold modeling of the PDE3A-SLFN12 complex with a partial 43S pre-initiation complex predicted binding near eIF3B and the eIF2αβγ-tRNA ternary complex at the mRNA entry channel. By mapping the interactomes surrounding the drug-induced complex, our study provides important mechanistic context for how PDE3A modulators act within the cell. In our proposed model, anagrelide remodels the interactome of the PDE3A-SLFN12 complex, which relocalizes and accumulates near the mRNA entry channel of the 43S pre-initiation complex.
    DOI:  https://doi.org/10.1016/j.mcpro.2026.101663
  14. Adv Sci (Weinh). 2026 Sep 16. e77822
      Tau aggregation is a central pathological feature of Alzheimer's disease, yet how different forms of tau-ranging from monomers to small soluble aggregates and mature fibrils-interact with the cellular environment remains poorly understood. Here, we combine immunoaffinity proteomics with single-molecule techniques and super-resolution microscopy to systematically map the tau interactome across defined aggregation states, spanning monomeric tau, nanoscopic soluble aggregates, and fibrillar species. Using post-mortem Alzheimer's disease brain tissue, we identify distinct functional modules associated with different aggregation states: while proteostasis factors and immune-related proteins preferentially associate with nanoscopic aggregates (oligomers), cytoskeletal, metabolic, and RNA-binding proteins are enriched for mature fibrillar tau. Single-molecule microscopy directly confirms this conformation-dependent recruitment for key interactors including Hsp70-2, ENO1, hnRNPA1, APP, EAAT4, and ubiquitin. A primary-neuron system with accelerated tau aggregation is used to model these findings in a controlled system, showing striking similarities to the brain samples. Finally, pseudotime analysis reconstructs a progressive remodelling of the tau interactome across disease progression, revealing stage-specific pathway vulnerabilities. Together, these results establish a temporally resolved framework for tau pathology shaped by protein interactions and identify potential therapeutic intervention points for investigation across stages of disease.
    Keywords:  Alzheimer's disease; Tau; interactome; proteomics; single‐molecule
    DOI:  https://doi.org/10.1002/advs.77822
  15. Handb Exp Pharmacol. 2026 Sep 13.
      Targeted protein degradation (TPD), particularly proteolysis-targeting chimeras (PROTACs), has emerged as a powerful strategy for eliminating disease-associated proteins through the ubiquitin-proteasome system. However, current PROTACs rely heavily on a limited number of E3 ligases, mainly CRBN and VHL, due to the scarcity of suitable ligands. This review summarizes the development of classical small-molecule E3 ligase ligands and highlights emerging biological recruitment strategies involving antibodies, nanobodies, aptamers, peptides, and engineered proteins. These approaches expand the accessible E3 ligase landscape, broaden the range of targetable proteins, and provide greater programmability and selectivity. Recent advances in delivery systems, modular activation, and artificial intelligence-assisted design are also discussed. Finally, keytranslational challenges, including intracellular delivery, immunogenicity, pharmacokinetics, manufacturing scalability, and off-target effects, are considered. Together, these advances provide a broader framework for developing more precise and versatile next-generation targeted protein degradation platforms.
    DOI:  https://doi.org/10.1007/164_2026_842
  16. Sci Adv. 2026 Sep 18. 12(38): eaeh0227
      The 26S proteasome engages with ubiquitinated substrates primarily through its constituent ubiquitin (Ub) receptors, which initiates a cascade of proteolytic processes. Leveraging this recognition mechanism, we developed a targeted protein degradation (TPD) strategy that recruits substrates directly to the proteasome, thereby bypassing the ubiquitination step. Our proteasome-targeting chimera, Protea-Tac, is a heterobifunctional protein degrader composed of a Ub receptor and an intracellular antibody. This chimera integrates into 26S proteasomes without altering their structural or functional integrity. Protea-Tac with cognate antibodies degraded various target proteins, including c-Fos, BRD4, FlagTDP43, HAtau, and GFPODC. We mechanistically demonstrated that this platform is (i) modular, allowing facile target switching, (ii) Ub independent, and (iii) highly target specific. Furthermore, Protea-Tac exhibited potent in vivo antitumor efficacy, posttranslationally inducing c-Fos degradation and substantially delaying tumor progression through both viral and nonviral delivery systems. These findings identify Protea-Tac as a distinct TPD platform capable of directly degrading intracellular proteins via engineered 26S proteasomes.
    DOI:  https://doi.org/10.1126/sciadv.aeh0227
  17. Cell Chem Biol. 2026 Sep 14. pii: S2451-9456(26)00321-1. [Epub ahead of print]
      Neurons are highly specialized cells whose function depends on dynamic regulation of gene expression. With increasing subcellular compartmentalization, neuronal gene expression becomes less dependent on transcription in the perinuclear region and tends to be increasingly controlled by local translation. The integrated stress response is a central, evolutionarily conserved pathway of translational regulation that maintains neuronal homeostasis through precise control of protein synthesis. Transient cellular stress, such as neuronal infection, adaptively activates this pathway, whereas persistent dysregulation, as observed in neuroinflammation and neurodegeneration, promotes neuronal dysfunction and damage. Here, we summarize key neuron-specific mechanisms governing translational homeostasis and discuss adaptive and detrimental molecular processes associated with neurological disease. Understanding this tightly regulated pathway and its context-dependent effects provides a framework for identifying mechanisms of neuronal vulnerability and for developing therapeutic strategies that target translational control in neurological disorders, while preserving its essential adaptive functions under physiological and acute stress conditions.
    Keywords:  CNS infection; integrated stress response; neurodegeneration; neuroinflammation; proteostasis; translational control
    DOI:  https://doi.org/10.1016/j.chembiol.2026.08.011
  18. Nat Commun. 2026 Aug 13. pii: 9751. [Epub ahead of print]17(1):
      Attachment of the ubiquitin-like protein (UBL) FAT10 onto substrates targets them for proteasomal degradation. Like ubiquitin, FAT10 is activated by the E1 enzyme UBA6 then transferred to E2 enzymes, but mechanisms controlling ubiquitin versus FAT10 activation by UBA6 and FAT10 transfer onto E2s remain unclear. Using cryo-EM, we visualise all stages of FAT10 E1-E2 handover: adenylation, thiolation and transthiolation. We find that FAT10 monopolises UBA6 by out-competing ubiquitin for thiolation and blocking the adenylation domain, preventing further UBL recruitment and promoting FAT10 signalling. We profiled UBA6-compatible E2 enzymes and found FAT10 transfer is restricted to a select subset associated with specific cellular pathways. UBE2Z (USE1) showed highest activity followed by UBE2D2, UBE2J2 and UBE2S. Capturing FAT10 or ubiquitin transfer from UBA6 to UBE2Z reveals UBE2Z is highly specialised for FAT10 transfer. It simultaneously engages both FAT10 domains (UBL1 and UBL2) and co-ordinates the metabolite inositol hexakisphosphate (InsP6) bound within the UBA6 catalytic domain. This InsP6 co-ordination extends to other FAT10 compatible E2s. Together, our structural and biochemical analyses reveal regulatory mechanisms underpinning FAT10 activation and transfer. We define principles governing selective FAT10 transfer, highlighting favourable interactions with FAT10 C-terminal domain (UBL2) and stable UBA6 binding, ensuring controlled conjugation onto substrates.
    DOI:  https://doi.org/10.1038/s41467-026-76603-3
  19. Trends Pharmacol Sci. 2026 Sep 14. pii: S0165-6147(26)00202-6. [Epub ahead of print]
      Targeted protein degradation has emerged as a powerful way to eliminate disease-driving proteins rather than merely inhibit them. However, most current approaches depend on ubiquitin ligases, whereas midnolin bypasses E3 recruitment and substrate ubiquitination while converging on the same 26S proteasome used for canonical ubiquitin-dependent degradation. Recent structural, biochemical, and disease studies have defined how midnolin recognizes β-strand-forming degrons, positions substrates at the 26S proteasome, and contributes to interferon regulatory factor 4 regulation, while engineered midnolin-targeting chimeras and midnolin-based targeting chimeras have redirected this pathway toward β-catenin and c-Myc. By linking endogenous pathway biology with engineered degrader pharmacology, this review compares midnolin-directed degradation with proteolysis-targeting chimeras and molecular glue degraders and identifies the design, delivery, and safety principles that will determine whether it becomes a useful ubiquitin-independent complement to existing degraders.
    Keywords:  MbTAC; MidTAC; TPD; midnolin–proteasome pathway; ubiquitination-independent proteasomal degradation
    DOI:  https://doi.org/10.1016/j.tips.2026.08.005
  20. Nat Commun. 2026 Aug 14. pii: 9775. [Epub ahead of print]17(1):
      Proteolytic processing is a fundamental regulatory mechanism in eukaryotic cells, yet the molecular identities and mechanisms underlying such events are often poorly defined. Silencing Defective 2 (SDE2), an essential human protein, plays important roles in mRNA splicing, DNA repair and ribosomal biogenesis. Cleavage of SDE2 downstream to its N-terminal ubiquitin-like domain (SDE2UBL) releases the biologically functional C-terminal domain (SDE2CT), highlighting the importance of this proteolytic event. However, the protease responsible for this cleavage in human cells has remained undefined. Here, we identify deubiquitinating enzyme, ubiquitin-specific protease 5 (USP5), as the selectively primary effector of SDE2 cleavage both in vitro and in cell. Biophysical and structural analysis suggests that SDE2UBL engages with USP5 through a two-site interaction that mirrors key features of ubiquitin recognition, supporting a mechanism of substrate mimicry. Functionally, depletion of USP5 increases intron retention in previously reported SDE2-dependent transcripts, linking the removal of SDE2UBL domain by USP5 to the role of SDE2 in mRNA splicing. Together, these findings reveal a non-canonical proteolytic function of USP5, uncovering a previously unrecognised regulatory axis linking deubiquitinating enzymes to protein maturation, expanding the substrate repertoire for USP5 and providing a framework for identifying protease-substrate relationships in post-translational regulation.
    DOI:  https://doi.org/10.1038/s41467-026-76751-6
  21. Trends Biochem Sci. 2026 Sep 14. pii: S0968-0004(26)00272-0. [Epub ahead of print]
      Cellular dormancy requires ribosomes to be reversibly silenced yet preserved. Gluc et al. identify Shwachman-Bodian-Diamond syndrome domain-containing hibernation factor (SNOR), a fission yeast hibernation factor induced specifically by perturbed glucose homeostasis. Rather than driving translational shutdown, SNOR maintains dormant ribosomes in a state competent for rapid reactivation.
    Keywords:  hibernation; mRNA; ribosome; stress; translation
    DOI:  https://doi.org/10.1016/j.tibs.2026.08.009
  22. STAR Protoc. 2026 Sep 17. pii: S2666-1667(26)00499-5. [Epub ahead of print]7(4): 104846
      Receptor endocytosis and lysosomal delivery are critical for efficient intracellular delivery of antibody-drug conjugates (ADCs), yet many cancer targets exhibit limited internalization. Here, we present a protocol for engineering and evaluating ADCs to induce receptor ubiquitination, thereby promoting receptor uptake and transport toward lysosomal compartments. We describe steps for designing antibodies fused with E3 ligase-binding modules, drug conjugation, and cellular assays to quantify ubiquitination-dependent internalization and lysosomal accumulation in cancer cell models. For complete details on the use and execution of this protocol, please refer to Zhuang et al.1.
    Keywords:  Biotechnology and bioengineering; Flow Cytometry; Microscopy; Protein expression and purification; SPR; Surface plasmon resonance; antibody
    DOI:  https://doi.org/10.1016/j.xpro.2026.104846
  23. Nat Commun. 2026 Aug 20. pii: 9945. [Epub ahead of print]17(1):
      Recent advances in de novo protein design have greatly outpaced standard protein biochemistry workflows, making experimental validation a bottleneck. Here, we describe workflows to address the scale, speed and reproducibility of common in vitro protein testing methods, enabling at least an order of magnitude increase in throughput while reducing wetlab time. Semi-Automated Protein Production (SAPP) is a rapid, modular, scalable and cost-effective protocol, enabling up to milligram-scale protein production and standardized characterization - including yield, dispersity, and oligomeric state - of hundreds of designs per day, at the cost-equivalent of a few DNA oligos per construct. End-to-end protocol execution takes 48 hours, with ~6 hours spent benchside using standard laboratory equipment. We showcase the platform by rapidly screening redesigned fluorescent proteins, as well as identifying de novo binders that potently neutralize respiratory syncytial virus. We also developed a barcoding and demultiplexing protocol (DMX) to further reduce gene synthesis cost 5-fold by leveraging oligo pools as input DNA for the generation of thousands of sequence-verified arrayed clones. These protocols which combine optimized molecular biology, automated analysis, and optional open-source robotics should be widely adoptable, accelerating protein design.
    DOI:  https://doi.org/10.1038/s41467-026-76740-9
  24. J Cell Biol. 2026 Nov 02. pii: e202508151. [Epub ahead of print]225(11):
      The Golgi apparatus is essential for protein secretion and cellular homeostasis, yet its organization and turnover in living tissues remain poorly understood. Golgiphagy, the selective autophagic degradation of the Golgi, has emerged as a key quality control pathway, but its physiological regulation in vivo remains largely unknown. Here, we generated two reporter mouse lines for constitutive or Cre-dependent visualization of Golgi architecture and Golgiphagy, enabling quantitative single-cell analysis across tissues. These models revealed striking cell type- and tissue-specific heterogeneity in basal Golgiphagy, including higher activity in renal proximal than distal tubules and in cerebellar than cortical neurons. Starvation and lipopolysaccharide-induced inflammation also promoted Golgi remodeling and Golgiphagy in multiple organs, particularly the liver, spleen, and kidney. Together, these reporter mice provide a powerful genetic toolkit for studying Golgi dynamics in vivo and establish that Golgiphagy is a spatially heterogeneous and stress-responsive process under physiological conditions.
    DOI:  https://doi.org/10.1083/jcb.202508151
  25. ACS Chem Biol. 2026 Sep 18. 21(9): 2157-2168
      Targeted protein degradation (TPD), including proteolysis targeting chimeras (PROTACs) and molecular glue degraders (MGDs), is a promising therapeutic approach. However, systematic discovery of such small molecules remains a major challenge. Here, we present PhenoDEL, a novel phenotypic DNA-encoded library (DEL) screening platform that integrates one-bead one-compound DEL (OBOC-DEL) with the Beacon optofluidic system for single-cell analysis. By coculturing individual OBOC-DEL beads and engineered reporter cells in nanoliter-scale chambers, PhenoDEL enables time-resolved, single-cell phenotypic evaluation and direct linkage between compound identity and intracellular response. As a proof-of-concept, we demonstrate discrimination of active and inactive on-bead compounds using an FKBP12F36V-EGFP degradation reporter in PC-3 cells, followed by DNA barcode decoding.
    DOI:  https://doi.org/10.1021/acschembio.6c00234
  26. Proc Natl Acad Sci U S A. 2026 Sep 22. 123(38): e2622424123
      The transmembrane 6 superfamily (TM6SF) comprises two members: TM6SF1, a ubiquitously expressed lysosomal membrane protein of unknown function, and TM6SF2, an endoplasmic reticulum protein required for bulk lipidation of Apolipoprotein B-containing lipoproteins. Here, we used cryo-electron microscopy (cryo-EM) to determine the structure of human TM6SF1 at 2.9-Å resolution. TM6SF1 forms a polytopic homodimer, with each protomer comprising 10 transmembrane helices (TMs). TMs 1-6 form a pocket that accommodates a cholesterol molecule. Cell-based assays revealed that loss of TM6SF1 perturbs mTORC1 signaling, resulting in reduced phosphorylation of S6 kinase 1 and 4E-BP1 and constitutive activation of transcription factor EB (TFEB), and that cholesterol is required for these effects. Biochemical analyses support the model that TM6SF1 directly engages LAMTOR1, a component of Ragulator complex, in a cholesterol-dependent manner. Together, these findings identify TM6SF1 as a lysosomal cholesterol binding protein involved in regulating mTORC1 signaling.
    Keywords:  LAMTOR1; TM6SF1; cryo-EM; mTORC1; transcription factor EB
    DOI:  https://doi.org/10.1073/pnas.2622424123
  27. J Virol. 2026 Sep 14. e0128226
      Viruses have evolved a myriad of strategies to manipulate their host's protein synthesis machinery, studies of which uncovered many founding principles of translational control. Decades of research have established our now-extensive understanding of how these pathogens target the array of translation factors that recruit ribosomes and regulate scanning, start site selection, decoding, and termination. Meanwhile, the ribosome itself was largely regarded as a code-reading machine that lacks regulatory influence. Recent advances in cryo-electron microscopy and quantitative proteomics have upended this viewpoint by revealing the remarkable conformational and compositional plasticity that gives rise to functionally diverse and translationally selective ribosome populations in mammalian cells. Here, we discuss how viruses are once again at the forefront of this emerging field, often bypassing or rewiring host translation factors while directly controlling the ribosome's composition and intrinsic motions to selectively promote their non-canonical modes of translation. We offer cautionary perspectives on extra-ribosomal functions of ribosomal proteins, while highlighting the emergence of the 40S head domain as a dynamic platform targeted to control various translation processes, ranging from initiation to frameshifting, that expand viral coding capacity. Overall, we chart the rise of the ribosome from a passive player to an active regulatory hub at the center of viral translational control strategies.
    Keywords:  cryo-EM; ribosomal structure; ribosome; translational control; virus
    DOI:  https://doi.org/10.1128/jvi.01282-26
  28. Cell Discov. 2026 Sep 15. pii: 66. [Epub ahead of print]12(1):
      Beta-coronavirus infection disrupts endoplasmic reticulum (ER) homeostasis; however, the mechanisms by which viral proteins manipulate ER-resident factors remain unclear. Here, we report that the SARS-CoV-2 accessory protein ORF3a binds to the ER chloride channel CLCC1, thereby impairing ER ion homeostasis, activating the unfolded protein response, and inducing endomembrane remodeling. Using newly developed ratiometric reporters, we showed that this interaction exacerbated the basal levels of ER-phagy and nucleophagy. Importantly, CLCC1 counteracted ORF3a by sequestering it within the ER, attenuating its toxicity and suppressing viral replication, which established CLCC1 as a host restriction factor. We further elucidated a spatiotemporal mechanism: during early infection, ORF3a accumulates in the ER, co-assembles with CLCC1 into puncta, and disrupts ER homeostasis; at the later stage, ORF3a overload enables its escape from CLCC1-mediated ER retention and subsequent translocation to lysosomes, facilitating viral egress. This ER-centric function is conserved across diverse beta-coronaviruses, including SARS-CoV-1 and bat- or pangolin-derived strains, revealing a unified pathogenic strategy. Furthermore, in mouse brains, the ORF3a-CLCC1 axis recapitulates key neuropathological features, including ER stress, autophagic dysfunction, neuronal death, and neuroinflammation, providing a mechanistic link to COVID-19-associated neurological symptoms. Our findings identify CLCC1 as an essential host defense protein and establish a conceptual framework to guide future research on antiviral responses at the organelle level and related disease mechanisms.
    DOI:  https://doi.org/10.1038/s41421-026-00918-0
  29. J Biol Chem. 2026 Sep 16. pii: S0021-9258(26)02429-4. [Epub ahead of print] 113557
      Abnormal protein-protein interactions (PPIs) involving signaling proteins contribute to numerous human diseases. Targeting such aberrant PPIs with selective inhibitory molecules is a promising therapeutic strategy, yet assessing how many PPIs are affected by a candidate drug, and whether inhibition occurs before or after complex assembly, remains technically challenging. Here, we present a live-cell methodology that captures drug-sensitive PPIs by integrating drug-induced cellular responses with interaction mapping. As a proof of concept, we identify ERK1 PPIs that are selectively altered by a novel chemical molecule exhibiting anti-tumor efficacy. We further apply complementary real-time imaging tools to visualize the dissociation of pre-assembled ERK1/cofactor complexes in living cells. Our approach provides a generalizable framework for decoding drug activity on PPIs for virtually any protein target, enabling more precise and physiologically relevant evaluation of therapeutic mechanisms.
    Keywords:  BiFC; Cell-PCA; EI52; MAPK; U0126; protein-protein interaction; splitFAST2
    DOI:  https://doi.org/10.1016/j.jbc.2026.113557
  30. Nat Commun. 2026 Aug 13. pii: 9750. [Epub ahead of print]17(1):
      Megakaryocytes (MKs) are polyploid cells that maintain bone marrow homeostasis by secreting cytokines, including transforming growth factor β1 (TGFβ1). During neoplastic transformation, MKs accumulate in the bone marrow, promoting fibrotic remodeling that leads to myelofibrosis. However, the therapeutic potential of targeting MK cytokine secretion remains poorly understood. Because unconventional secretion of TGFβ1 and interleukin 1β (IL1β) via secretory autophagy occurs in other cell types, we investigated whether MKs may utilize the same mechanism. Disrupting secretory autophagy, or inhibiting the small GTPase RhoA or its downstream effector Rho-associated kinase (ROCK), markedly reduced TGFβ1 and IL1β secretion in vitro. Conditional deletion of the autophagy gene Atg5 in the hematopoietic system limited megakaryocytosis and aberrant cytokine secretion in an MPLW515L-driven transplant model, while MK-specific deletion of Rhoa protected mice from fibrosis. Ultimately, ROCK inhibition, alone or combined with a JAK2 inhibitor, also attenuated disease hallmarks, identifying the RhoA-secretory autophagy axis as a promising therapeutic target in myelofibrosis.
    DOI:  https://doi.org/10.1038/s41467-026-76615-z
  31. Nat Commun. 2026 Aug 14. pii: 9772. [Epub ahead of print]17(1):
      Phosphorylation-targeting chimeras (PhosTACs) enable targeted protein dephosphorylation by recruiting phosphatases through induced proximity. However, the direct recruitment of phosphatase subunits or holoenzymes with small molecules remains challenging, as suitable ligands are scarce and often compromise enzymatic activity or cellular function. Here, we present togoPhosTAC, a hybrid modality that integrates a small-molecule PhosTAC, an engineered FKBP12F36V-phosphatase, and a lipid nanoparticle delivery system. This strategy allows delivery of preassembled PhosTAC-FKBP12F36V-phosphatase complexes or PhosTAC-phosphatase mRNA, enabling rapid and efficient intracellular dephosphorylation. We demonstrate that togoPhosTAC can selectively dephosphorylate EGFR, α-synuclein, and tau in biological contexts, providing a versatile strategy that circumvents the need for genetically engineered phosphatases. We also find togoPhosTAC further enhances tau dephosphorylation as well as its disaggregation in cellulo. Importantly, intrahippocampal or intranasal delivery of togoPhosTAC in PS19 tau transgenic male mice leads to a marked reduction in pathological tau phosphorylation across multiple sites (Ser202, Thr205, Thr231, Ser396, and Ser404), decreases pathological tau burden in related brain regions, and improves Alzheimer's disease-related behavioral deficits. Together, these findings establish a versatile and generalizable approach for precise protein dephosphorylation in disease-relevant systems, overcoming key limitations in phosphatase-recruiting drug discovery.
    DOI:  https://doi.org/10.1038/s41467-026-76725-8
  32. Nat Commun. 2026 08 19. pii: 9900. [Epub ahead of print]17(1):
      Lipid droplets are dynamic cellular organelles that store neutral lipids and coordinate metabolic and stress-response pathways. In the brain, lipid droplets in glial cells, including astrocytes, have been implicated in Alzheimer's disease, but how genetic risk factors influence their composition and turnover remains poorly understood. APOE is the strongest genetic modulator of late-onset Alzheimer's disease and exists in common variants that confer decreased, neutral, or increased risk. Here we show that APOE genotype shapes the lipid droplet proteome, lipidome, and degradation dynamics in human induced pluripotent stem cell-derived astrocytes. By comparing oleic acid-treated astrocytes carrying APOE2, APOE3, or APOE4, we find that each variant is associated with distinct lipid droplet proteins and lipids. These molecular differences correspond to genotype-dependent changes in lipophagy, an autophagy-mediated pathway for lipid droplet clearance. Lipid droplets in APOE2 astrocytes undergo efficient autophagic turnover, whereas those in APOE4 astrocytes resist degradation. These findings identify impaired lipid droplet clearance as a potential mechanism linking APOE4 to Alzheimer's disease risk.
    DOI:  https://doi.org/10.1038/s41467-026-76565-6
  33. Protein Sci. 2026 10;35(10): e70787
      Expanding the repertoire of usable E3 ubiquitin ligases remains a critical challenge in the field of targeted protein degradation. For the emerging E3 ligase Kelch domain-containing protein 2 (KLHDC2), currently reported ligands have a carboxylate moiety in common that mimics the natural degron but compromises cellular permeability. Here we report the discovery of carboxylate-free ligands for KLHDC2 through high-throughput screening and cellular functional evaluation. A fluorescence polarization-based screening assay identified NL1 as a micromolar KLHDC2 binder, which was confirmed by orthogonal biophysical methods. Although the structure-activity study did not improve affinity, a degron reporter assay demonstrated measurable cellular target engagement. Encouraged by these results, an expanded screen further identified NL2 as a neutral ligand with improved cellular activity. Computational analysis suggests that NL2 adopts a distinct binding mode, in which the conformational flexibility of two loops of KLHDC2 enables accommodation of its aromatic scaffold, while hydrophobic and π-driven interactions compensate for the canonical ionic contacts of other carboxylate-containing ligands. This work demonstrates that KLHDC2 can accommodate non-carboxylate scaffolds and expands the chemical space for developing KLHDC2-based targeted protein degradation strategies.
    Keywords:  C‐degron pathway; E3 ubiquitin ligase; Kelch domain‐containing protein 2; high throughput screening; neutral ligand; targeted protein degradation
    DOI:  https://doi.org/10.1002/pro.70787
  34. PLoS Pathog. 2026 Sep 18. 22(9): e1014196
      The GID/CTLH E3 ligase complex is implicated in several biological processes, yet its full substrate repertoire remains poorly defined. We recently identified the complex as a broad modulator of macrophage responses to Mycobacterium tuberculosis (Mtb) infection. Here, we use label-free proteomics and diGly capture analysis of Mtb-infected macrophages to define the GID/CTLH-dependent ubiquitylome. We identify thousands of dynamically altered ubiquitylation sites, with strong enrichment among proteins involved in cellular metabolism and innate immune signaling. Concurrent proteome analysis revealed extensive rewiring in GID/CTLH-deficient macrophages, with >90% of enriched pathways among increased proteins consisting of metabolic targets. Notably, inhibitory phosphatases (PTEN, INPP5D) also emerged as candidate substrates. Functional studies revealed proteasome-dependent stabilization of PTEN and INPP5D in GID/CTLH-deficient macrophages with each phosphatase individually exerting an influence on Mtb intracellular survival. Together, our study defines a GID/CTLH-dependent ubiquitylome in macrophages and identifies the complex as a central regulator of metabolism and antimicrobial immunity.
    DOI:  https://doi.org/10.1371/journal.ppat.1014196
  35. J Cell Sci. 2026 Sep 14. pii: jcs.265223. [Epub ahead of print]
      The endoplasmic reticulum (ER) extends throughout neurons and regulates many functions, including neurite outgrowth, neurotransmission, and synaptic plasticity. Mutations in ER-shaping proteins cause the neurodegenerative disorder Hereditary Spastic Paraplegia (HSP), yet the ultrastructure and dynamics of neuronal ER remain largely unexplored, especially at presynaptic terminals. Using super-resolution and live imaging in Drosophila larval motor neurons, we investigated ER structure in wild-type animals and mutants of the HSP-linked gene, Atlastin, which encodes an ER-shaping protein. Previous studies reported diffuse localization of an ER luminal marker at Atlastin mutant presynaptic terminals, which was attributed to ER fragmentation. Using an ER membrane marker, we found that Atlastin mutant ER forms robust networks with only mild defects in structure and dynamics, indicating that the primary defect is functional rather than architectural. We demonstrate that Atlastin mutants progressively displace a luminal ER protein reporter to the cytosol during larval development, specifically at synapses, while this reporter remains correctly localized in cell bodies, axons, and muscles. This synapse-specific displacement phenotype, previously unreported in non-neuronal cells, emphasizes the importance of studying neurons to understand HSP pathogenesis.
    Keywords:   Drosophila ; Atlastin; Endoplasmic Reticulum; Synapse
    DOI:  https://doi.org/10.1242/jcs.265223
  36. J Biol Chem. 2026 Sep 15. pii: S0021-9258(26)02428-2. [Epub ahead of print] 113556
      Protein O-glucosyltransferase (POGLUT1/Rumi) transfers an O-linked glucose (O-Glc) monosaccharide from UDP-Glc to a serine residue in epidermal growth factor-like (EGF) repeats with a specific consensus sequence. In mammals, GXYLT1 and GXYLT2 transfer xylose to this O-Glc monosaccharide. Previous studies showed that genetic deletion of Poglut1 in mice leads to embryonic lethality with defects in Crumbs2 (CRB2) trafficking and NOTCH1 signaling during development. Both CRB2 and Notch receptors have multiple EGF repeats with the O-Glc modification site. However, the roles of xylosyl elongation of O-Glc in protein trafficking and mammalian embryonic development are unknown. Here, we demonstrated that xylosyl elongation of O-Glc occurs in the ER and comprehensively analyzed O-Glc glycosylation on CRB2, NOTCH1, and NOTCH2 expressed in HEK293T cells by mass spectrometry. We found that most EGF repeats containing the consensus sequence were modified with O-Glc glycans, while the degree of xylosyl elongation varied among different EGF repeats, suggesting EGF repeat-specific regulation. GXYLT1 knockout cells showed reduced or lost xylosyl elongation, but GXYLT2 knockout cells did not. Cell-based assays demonstrated decreased secretion of substrate proteins in GXYLT1 knockout cells, which was rescued by wild-type GXYLT1. Analysis of Gxylt1 and Gxylt2 knockout mice revealed that Gxylt1 deletion, but not Gxylt2 deletion, caused embryonic lethality. Altogether, our data suggest that GXYLT1 plays a major role in xylosyl elongation of O-Glc glycans and thereby contributes to the ER quality control and trafficking of CRB2, NOTCH1, and NOTCH2 in HEK293T cells while GXYLT1 and GXYLT2 have distinct biological functions during mouse embryonic development.
    Keywords:  Crumbs; EGF repeats; ER quality control; Endoplasmic reticulum; Notch; O-glucosylation; glycosylation; glycosyltransferase; mass spectrometry; post-translational modification; xylose
    DOI:  https://doi.org/10.1016/j.jbc.2026.113556
  37. Nat Commun. 2026 09 15. pii: 9788. [Epub ahead of print]17(1):
      Tumor progression is driven by cancer cells' ability to establish a cellular network through tunneling nanotube-like connections (TNTs), which enable mitochondrial exchange both within the tumor cells and with the tumor microenvironment (TME). However, the functional consequences of mitochondrial transfer between tumor and non-tumor cells, and its occurrence in vivo, remain poorly understood. Here we show bidirectional mitochondrial transfer between Glioblastoma (GBM) cells and non-tumoral astrocytes (AS). We report that transfer of damaged mitochondria from GBM cells to AS is associated with activation of mitophagy in recipient cells, while astrocyte-derived mitochondria to GBM cells correlates with changes in mitochondrial activity and metabolic readouts. Furthermore, intravital subcellular microscopy (ISMic) in a live animal model allows the visualization of TNT connections with characteristics similar to those observed in vitro and supported TNT-mediated mitochondrial transfer in vivo. These findings reveal a potential mechanism of tumor adaptation and highlight TNTs as promising therapeutic targets.
    DOI:  https://doi.org/10.1038/s41467-026-76619-9
  38. Nat Commun. 2026 Aug 20. pii: 9957. [Epub ahead of print]17(1):
      Metabolic reprogramming is a defining feature of cancer; however, how it contributes to therapeutic resistance remains incompletely understood. Here we show that loss of aldo-ketoreductase 1A1 (AKR1A1) in renal cell carcinoma (RCC) and hepatocellular carcinoma (HCC) disrupts terminal glycolytic flux and lactate production through S-nitrosylation-mediated inhibition of pyruvate kinase, resulting in the accumulation of methylglyoxal (MGO). In multiple AKR1A1-deficient models, but not in those endogenously expressing the C423/424 A mutant of pyruvate kinase M2, elevated MGO triggers autophagic degradation of Kelch-like ECH-associated protein 1, leading to Nuclear factor erythroid 2-Related Factor 2 (NRF2) activation and transcriptional reprogramming. This NRF2-driven response enhances chemoresistance and promotes tumor cell migration, two hallmarks of aggressive cancer. Therapeutically, we demonstrate that pharmacological inhibition of the glyoxalase system-the major pathway for MGO detoxification-restores drug sensitivity in patient-derived cells and xenograft models, revealing a context-dependent metabolic vulnerability in AKR1A1 loss conditions. These findings identify AKR1A1 as a metabolic tumor suppressor and uncover crosstalk between S-nitrosylation and glycation as a key regulatory axis linking metabolic reprogramming to NRF2-driven therapy resistance, offering glyoxalase inhibition as a potential precision treatment strategy for RCC and HCC.
    DOI:  https://doi.org/10.1038/s41467-026-76938-x
  39. Nat Commun. 2026 Sep 17. pii: 9883. [Epub ahead of print]17(1):
      In human cells, a subset of tRNA-encoding genes contain introns. These are removed by a spliceosome-independent pathway in which the tRNA splicing endonuclease complex catalyzes intron excision. The resulting exons are subsequently ligated by the tRNA-ligase complex (tRNA-LC), comprising Ashwin, CGI-99, FAM98B, DDX1, and RTCB/HSPC117. The molecular architecture and functions of its non-catalytic subunits remain poorly understood. Using cryo-EM, we determined an atomic-resolution structure of human tRNA-LC. CGI-99, DDX1, and FAM98B form an α-helical bundle that contacts RTCB opposite its active site and anchors DDX1 via its C-terminal helix. FAM98B and CGI-99 form an extensively co-folded heterodimer that clamps Ashwin in a pincer-like structure. Structure-based mutagenesis supports the architecture of the complex. We further show that FAM98A and FAM98C assemble distinct RTCB-containing complexes lacking Ashwin, suggesting specialized cellular functions. Our results provide insights into the molecular assembly of the tRNA ligase complex, highlighting its functions in tRNA biogenesis and beyond.
    DOI:  https://doi.org/10.1038/s41467-026-77450-y
  40. J Biol Chem. 2026 Sep 18. pii: S0021-9258(26)02452-X. [Epub ahead of print] 113580
      Medulloblastoma is the most common pediatric brain cancer, but current treatments are largely non-specific, often causing developmental side effects. Genomic sequencing identified the RNA helicase DDX3X as one of the most frequently mutated genes in this cancer and a potential treatment target, yet its role in tumor progression remains elusive. Prior studies have indicated that the mutations cause specific defects in translation; however, both DDX3X and its yeast ortholog Ded1 have also been associated with cellular stress responses, suggesting that the contribution of the DDX3X mutations to medulloblastoma might result from defects in the translational response to stress. Building on our prior study that replicated the DDX3X mutations in yeast DED1 (ded1-mam), we examined the mutants' effects following TOR pathway inactivation. First, we demonstrated that ded1-mam displayed substantial rapamycin-resistant growth compared to wild-type cells. Additionally, similar to other ded1 mutants, the ded1-mam had increased protein abundance of Ded1 and the translation factor eIF4G1 under TOR inactivation. Notably, these differences did not result in increased bulk translation following rapamycin; rather, the growth phenotypes appeared to be driven by translation of specific mRNAs. Reporter assays demonstrated enhanced translation of mRNA with an unstructured 5' UTR in ded1-mam following TOR inhibition and a decrease in a structured reporter. Furthermore, known Ded1 target genes with relatively unstructured 5' UTRs showed upregulated protein levels in rapamycin. We hypothesize that mutant DDX3X selectively upregulates translation of unstructured, pro-growth transcripts while downregulating other structured transcripts, allowing tumor cells to bypass stress-induced growth controls and promoting medulloblastoma progression.
    Keywords:  RNA helicase; Saccharomyces cerevisiae; cancer; eukaryotic translation initiation; medulloblastoma; stress response; target of rapamycin (TOR)
    DOI:  https://doi.org/10.1016/j.jbc.2026.113580
  41. Cell Chem Biol. 2026 Sep 15. pii: S2451-9456(26)00322-3. [Epub ahead of print]
      Identifying therapeutic protein targets is challenging due to unpredictable druggability. Exploring multi-purpose drug targetomes, such as that of rapamycin, offers a valuable strategy for target discovery. Here, using chemical proteomics, we identified the histone-binding protein RBBP7 as a previously unrecognized target of rapamycin. Rapamycin disrupts the RBBP7-H4 interaction, leading to reduced histone H4 acetylation and downregulation of DNMT1. Based on this mechanism, we designed RBBP7-targeting peptides (RBBP7-TPs). RBBP7-TP4 exhibited potent anti-tumor activity in both cellular and xenograft mouse models of hepatocellular carcinoma (HCC). Furthermore, RBBP7-TP4 synergized with HDAC inhibitors to enhance epigenetic efficacy in HCC models, supporting a promising combination strategy. Collectively, our findings highlight the value of mining multi-purpose drug targetomes for therapeutic target discovery. RBBP7 is established as a druggable target in cancer, and RBBP7-TP4 represents a potential peptide-based therapeutic candidate.
    Keywords:  HDAC inhibitors; RBBP7; chemical proteomics; drug target; rapamycin
    DOI:  https://doi.org/10.1016/j.chembiol.2026.08.012
  42. Nat Commun. 2026 Aug 20. pii: 9950. [Epub ahead of print]17(1):
      Protein arginine deiminases (PADs) convert arginine to citrulline, altering protein structure. Of the five human isozymes, PAD1-4 are catalytically active with distinct tissue-specificities, yet isozyme-specific substrate recognition remains poorly defined. Here, we perform in vitro lysate-based substrate profiling of PAD1-4 via mass spectrometry across human H4 and HeLa cell lines, identifying ~30,000 citrullination sites across ~5,500 proteins. Only 14% of sites are shared among all, reflecting distinct sequence preferences: PAD1-2 show broad specificity, whereas PAD3-4 favor arginines flanked by acidic or glycine residues. These preferences persist over 10 min-16 h, indicating sequence context rather than temporal dynamics drives specificity. Mutation analysis of eleven PAD4 variants reveal Q346, G403, R639, and H640 as key determinants distinguishing substrate recognition from that of PAD2. This work provides a comprehensive in vitro atlas of maximal substrate capacity, defining isozyme-specific motifs and molecular determinants to guide selective inhibitors and probes for citrullination mechanisms in health and disease.
    DOI:  https://doi.org/10.1038/s41467-026-76752-5
  43. Autophagy. 2026 Sep 16.
      Metabolism and autophagy are closely interconnected, but whether metabolic alterations can chemically modify proteins and influence their selective autophagic degradation remains poorly understood. Our recent findings show that increased S-nitrosylation resulting from the loss of the denitrosylase AKR1A1/SCoR impairs glycolysis and promotes methylglyoxal accumulation, leading to carbonyl modification and selective autophagic degradation of KEAP1. These findings suggest that metabolic rewiring can influence the fate of specific proteins. In particular, we discuss whether glycation may act as a metabolic "mark" for selective autophagy and how the removal of metabolically modified proteins may, in turn, sustain signaling pathways involved in tumor progression and therapy resistance.
    Keywords:  AKR1A1; KEAP1; NRF2; SCoR; cancer metabolism; glycation; glycolysis; methylglyoxal; nitric oxide; pyruvate kinase
    DOI:  https://doi.org/10.1080/15548627.2026.2735183
  44. Adv Sci (Weinh). 2026 Sep 13. e77305
      Targeted protein degradation (TPD) redirects endogenous protein-disposal pathways to selectively eliminate therapeutically relevant proteins. Over the past two decades, this field has progressed from proteolysis-targeting chimeras (PROTACs) to an expanding repertoire of proteasomal and lysosomal degradation strategies. Recent studies indicate that supramolecular strategies are emerging in TPD, with dynamic noncovalent interactions and ordered assembly shaping degrader construction, delivery, functional integration, or intracellular assembly or activation. Here we organize recent advances into four major modes of supramolecular involvement, encompassing noncovalent modular decoupling of degrader components, delivery-oriented single-component self-assembly, multicomponent co-assembly for functional integration, and intracellular in situ assembly that generates either persistent local architectures or active degraders following cellular entry. We discuss their applications across proteasomal, endosomal-lysosomal, and autophagy-lysosomal degradation pathways. Finally, we discuss translational challenges and emerging opportunities for advancing supramolecular degraders, spanning modular design, strategy diversification, indication selection, systemic biodistribution, intracellular trafficking, safety assessment, and the prospective use of artificial intelligence (AI)-assisted modeling.
    Keywords:  co‐assembly; degrader; self‐assembly; supramolecular chemistry; targeted protein degradation
    DOI:  https://doi.org/10.1002/advs.77305
  45. Adv Sci (Weinh). 2026 Sep 16. e77854
      Disuse-induced muscle atrophy is characterized by coordinated metabolic remodeling and enhanced protein degradation, yet the molecular link between these processes remains unclear. Here, we identify 6-phosphofructo-2-kinase/fructose-2,6-bisphosphatase 3 (PFKFB3) as a critical regulator of this condition. Using a unilateral hindlimb immobilization mouse model, metabolomics, and functional assays in vivo and in vitro, we demonstrate that PFKFB3 is markedly upregulated during muscle atrophy and promotes myofiber wasting. Mechanistically, PFKFB3 predominantly localizes to the nucleus and functions independently of its canonical glycolytic activity. It acts as a scaffold protein to facilitate the interaction between the E3 ubiquitin ligase Nedd4 and the transcription factor JunB, thereby enhancing JunB ubiquitination and proteasomal degradation. Loss of JunB, a known anti-atrophy factor, contributes to atrophic progression. In turn, JunB transcriptionally represses PFKFB3, forming a regulatory feedback loop. Pharmacological and genetic inhibition of the PFKFB3-Nedd4-JunB axis significantly attenuates muscle atrophy in vivo. Collectively, these findings reveal a noncanonical nuclear function of PFKFB3 in coordinating protein stability during muscle atrophy and highlight this signaling axis as a potential therapeutic target for disuse-induced muscle wasting.
    Keywords:  PFKFB3; disuse; muscle atrophy; nuclear translocation; ubiquitination
    DOI:  https://doi.org/10.1002/advs.77854
  46. Nat Commun. 2026 08 19. pii: 9915. [Epub ahead of print]17(1):
      TBK1 kinase is a central regulator of type I IFN production. Upon activation of the IFN-β induction pathway, TBK1-adaptor proteins (NAP1, SINTBAD, TANK) form liquid condensates. We show that NAP1 condensates concentrate TBK1. Using NAP1KO cell lines, we demonstrate that NAP1 exerts a dual effect on TBK1 activity. Initially, NAP1 binds TBK1 and increases its activity, promoting IFN pathway activation. Subsequently, TBK1-mediated phosphorylation of NAP1 induces the formation of condensates. These NAP1 condensates concentrate both TBK1 and the phosphatase PP2A, which dephosphorylates and consequently deactivates TBK1, thus limiting IFN induction. Additionally, in patients with lupus or interferonopathies, we identify NAP1 variants unable to form condensates upon danger signal exposure, which sustain TBK1 activation without limiting its activity. This study reveals a mode of regulating a signaling pathway through condensate formation and provides a potential molecular explanation for immune dysregulation associated with NAP1 variants in certain patients with interferonopathies.
    DOI:  https://doi.org/10.1038/s41467-026-76769-w
  47. Autophagy. 2026 Sep 16.
      Mitochondrial ubiquitination is a central component of mitochondrial quality control. The PINK1 (PTEN induced kinase 1)-PRKN (parkin RBR E3 ubiquitin protein ligase) pathway established how loss of mitochondrial membrane potential can trigger a phospho-ubiquitin feed-forward cascade on the outer mitochondrial membrane (OMM). It remains less clear how mitochondrial ubiquitination is achieved when PRKN is absent or inactivated. In our recent work, we identify a recruitment platform organized by AMBRA1 (autophagy and beclin 1 regulator 1), in which RMC1 (regulator of MON1-CCZ1) positions HUWE1 (HECT, UBA and WWE domain containing E3 ubiquitin protein ligase 1) at mitochondria. This spatial arrangement promotes HUWE1-dependent ubiquitination and turnover of OMM proteins, including MFN2 (mitofusin 2), VDAC1 (voltage-dependent anion channel 1), and VDAC2 (voltage-dependent anion channel 2). Our findings raise the question of how cells select among distinct mitochondrial ubiquitination pathways and whether these pathways function independently, sequentially, or cooperatively.
    Keywords:  AMBRA1; HUWE1; PINK1-PRKN; RMC1; mitochondrial quality control; ubiquitination
    DOI:  https://doi.org/10.1080/15548627.2026.2735210
  48. Proc Natl Acad Sci U S A. 2026 Sep 22. 123(38): e2622980123
      RNA is built from a four-nucleotide alphabet. Complementary sequences inevitably arise, creating pervasive opportunities for promiscuous RNA-RNA interactions. Here, we show that this chemistry makes the transcriptome intrinsically prone to self-association. We simulated the simultaneous interactions of ~7,500 mRNAs representing the Escherichia coli transcriptome at physiological concentrations. These large-scale simulations predict widespread, dynamic clustering driven by RNA alone and organized by long, multivalent transcripts. Purified mRNA recapitulates this behavior in vitro, with aggregate composition mirroring model predictions. Strikingly, native mRNA sequences are markedly less prone to self-association than matched randomized controls: They fold more stably, expose shorter single-stranded regions, and form weaker intermolecular contacts. Similar signatures are observed in abundant human mRNAs, suggesting that evolution has shaped coding sequences to minimize self-association. These findings identify transcriptome solubility as an unrecognized constraint on mRNA sequence evolution and provide a framework for understanding how cells keep their transcriptomes dispersed and functional.
    Keywords:  RNA aggregation; RNA–RNA interactions; biomolecular condensates; molecular evolution; transcriptome dynamics
    DOI:  https://doi.org/10.1073/pnas.2622980123
  49. PLoS Comput Biol. 2026 Sep 18. 22(9): e1014188
      Archaea represent one of the three domains of cellular life and yet account for fewer than 1% of experimentally determined protein structures, leaving the extent of their structural novelty unknown. Here we present a systematic domain-level classification of 124,075 proteins from 65 archaeal classes spanning 21 phyla and all major lineages, using both AFDB and newly predicted AlphaFold3 structures classified against the Evolutionary Classification of protein Domains (ECOD). Archaeal proteins span 987 of the 2,457 ECOD X-groups defined across all cellular life, roughly 40% of known fold diversity captured within a single domain of life. Clustering by Foldseek recovered structural relationships for 63% of domains that are singletons by sequence comparison. To characterize the 21% of proteins lacking high-confidence classification, we applied successive filters for structure prediction confidence, protein length, and structural cluster context, reducing 8,452 domain-free proteins to a small number of well-folded structural orphans (less than 0.1% of the dataset). The unclassified fraction is dominated by sub-threshold matches (matches below the 0.85 DPAM confidence cutoff for high-confidence T-group assignment) to known folds (14% of all proteins) and low-confidence structure predictions (5%), not by novel structures. These results demonstrate that the protein fold repertoire at the single-domain level is broadly conserved across the deepest phylogenetic distances in cellular life, and that the gap between archaeal and well-characterized proteomes reflects classification sensitivity for divergent sequences rather than unexplored structural diversity.
    DOI:  https://doi.org/10.1371/journal.pcbi.1014188
  50. PLoS Biol. 2026 Sep;24(9): e3003556
      Flaviviruses (genus Orthoflavivirus) are arthropod-borne viruses which cause approximately 400 million annual global infections in humans. Flavivirus infection requires cellular machinery to facilitate replication and spread. All known flaviviruses replicate in association with the host endoplasmic reticulum (ER), where genome replication is confined within virus-induced ER invaginations called viral replication organelles (vROs). Despite the central role of these structures during flavivirus infection, the mechanisms underlying vRO biogenesis remain undefined-particularly the membrane rearrangements required for their formation. In this work, we report a conserved role for a cellular ER remodeling protein, atlastin-2 (ATL2), in the organization of vROs within infected cells. Using confocal and electron microscopy, we show that ATL2 depletion leads to a reduction in vRO spatial distribution in flavivirus-infected cells. Changes in vRO distribution corresponded with a decrease in virus production and robust induction of innate immune responses. We also demonstrate that ATL2 accumulates in areas of vRO formation during flavivirus infection. Critically, mutational analysis showed that a tethering-competent but fusion-defective ATL2 mutant was sufficient to rescue DENV and ZIKV replication in ATL2-knockout cells. Finally, targeting of ATL2 activity using synthetic peptides significantly reduced DENV replication in both immortalized and human primary cells, suggesting a possible avenue for targeting host ER functions to limit flavivirus replication. Taken together, these results show that membrane tethering plays a critical and conserved role in flavivirus infection, functioning to organize membranes for vRO biogenesis and limit cellular immune activation. Importantly, we provide evidence that ATL2-mediated membrane organization can be targeted to inhibit viral replication.
    DOI:  https://doi.org/10.1371/journal.pbio.3003556
  51. J Biol Chem. 2026 Sep 16. pii: S0021-9258(26)02431-2. [Epub ahead of print] 113559
      In signaling cascades, signaling proteins often encode multiple domains or motifs, which presents the possibility for avidity -- where multivalent binding drastically increases interaction strength and duration. However, predicting and validating multivalent interactions that interact with avidity is a challenge. Here, we integrate mechanistic modeling, structure-based analysis, and experimental approaches as a framework for defining the conditions under which avidity plays a role. We explore the tandem SH2 domain family of interactions with bisphosphorylated partners as a multivalent archetype, which encompasses key secondary messengers in tyrosine kinase signaling networks. Theoretical modeling suggests that maximum avidity occurs with closely spaced tyrosine phosphorylation sites combined with moderate monovalent affinities - exactly around the innate range of SH2 domain affinity - or with phosphorylation sites separated by sufficiently flexible linkers. Surprisingly, despite sequence diversity, structure-based analysis showed relatively conserved three-dimensional spacing between SH2 domains across all tandem SH2 families, which we corroborate experimentally, suggesting evolutionary optimization for avidity interactions. The combination of structure-based analysis of domain spacing with available monovalent experimental data appears, along with iterative experimental refinement of biophysical parameters, can identify high affinity interactions of tandem SH2 domain recruitment to the EGFR C-terminal tail. Using these principles, we extended bivalent predictions into the full phosphoproteome space and structural parameterization of other partners of SH2 domain binding, providing resources and methods for more rapid expansion of bivalent analysis. These approaches lay the groundwork for larger utility in multivalent prediction and testing to help better understand protein interactions that drive cell signaling.
    Keywords:  BLI; SH2 domains; avidity; biolayer interferometry; bivalent; computational biology; phosphotyrosine; protein interactions
    DOI:  https://doi.org/10.1016/j.jbc.2026.113559
  52. Oncogene. 2026 Sep 16.
      CDC73 is a well-characterized tumor suppressor regulated by stress stimuli, governing progression of diverse human malignancies. Although previous studies have shown that E3 ubiquitin ligase UBR5 drives CDC73 ubiquitination and degradation to modulate tumorigenesis, the mechanisms by which stress-responsive pathways regulate UBR5-mediated CDC73 inactivation and transcriptional reprogramming remain elusive. Here, via integrated analyses of public datasets, multi-omics profiling (assay for transposase-accessible chromatin with sequencing [ATAC-seq], cleavage under targets and tagmentation [CUT&Tag], mRNA sequencing [mRNA-seq]), in vitro/in vivo assays, and molecular approaches including co-immunoprecipitation (Co-IP) and molecular docking, we demonstrate that UBR5 depletion profoundly alters chromatin accessibility and genome-wide transcriptional profiles in a CDC73-dependent manner. UBR5 ablation markedly suppresses osteosarcoma malignant phenotypes in cultured cells and xenograft models, with these effects fully rescued by concurrent CDC73 silencing. Mechanistically, we identify the JNK cascade as the critical upstream regulator: JNK activation sustains CDC73 stability by antagonizing UBR5-mediated CDC73 polyubiquitination, and map Lys257 as the key residue for UBR5-dependent CDC73 ubiquitination and degradation. Collectively, our findings define a novel JNK-dependent UBR5-CDC73 axis that acts as a molecular brake of the CDC73 positive feedback loop to orchestrate transcriptional programs, providing new mechanistic insights into CDC73 post-translational regulation in tumorigenesis and promising therapeutic targets for CDC73-dysregulated diseases.
    DOI:  https://doi.org/10.1038/s41388-026-03978-4
  53. Nat Commun. 2026 Aug 20. pii: 9964. [Epub ahead of print]17(1):
      Alternative tandem transcription initiation is a pervasive mechanism of gene regulation, yet its genetic impact on human disease remains largely unknown. Here, we systematically quantify the genetic regulation of alternative tandem transcription initiation across 25,859 samples from 49 normal human tissues and 33 tumor tissues. We identify approximately 0.4 million genetic variants associated with alternative transcription initiation in 5295 genes, with 32% operating independently of gene expression. Moreover, we discover 2238 multi-tissue alternative tandem transcription initiation outliers enriched for rare deleterious promoter and 5' UTR variants, demonstrating that both common and rare variants modulate transcription initiation. Strikingly, 74% of disease variants that colocalize with genetic variants regulating alternative transcription initiation cannot be identified through expression quantitative trait loci. Transcriptome-wide association studies identify 614 disease susceptibility genes associated with alternative transcription initiation, including known cancer drivers such as MAFF and MLLT10. Functional validation uncovers OSGEP as a breast cancer risk gene, where the alternative allele lengthens the 5' UTR and reduces protein abundance through upstream open reading frame-mediated translation repression, and suppresses breast cancer cell proliferation. Our findings establish alternative transcription initiation as a major, underappreciated mechanism associating noncoding variation with disease, providing a critical resource for interpreting disease risk loci.
    DOI:  https://doi.org/10.1038/s41467-026-76741-8
  54. G3 (Bethesda). 2026 Sep 16. pii: jkag245. [Epub ahead of print]
      The proteasome is essential for proteostasis. Transcriptional induction of proteasomal components occurs when the proteasome is inhibited, but an overview of the transcriptional responses caused by proteasome perturbation is missing. Here, we profiled transcriptional changes caused by chemical and genetic proteasome inhibition and defined time-dose responses in cells and organoids. Induction of proteasome components varied by cell type and inhibition mode, whereas other responses were consistent, including upregulation of chaperones and secreted factors, and repression of cell cycle regulators. A proteasome stress response signature was defined based on the genes consistently modulated across systems, and applying this signature to aging datasets revealed activation of this stress response in some tissues, including skeletal muscle. Moreover, secreted factors within the signature showed similar age-related changes in human plasma, suggesting systemic activation of this stress response with aging. Together, these findings define a transcriptional signature for monitoring proteasome stress during aging and age-related diseases.
    Keywords:  aging; organoids; proteasome; proteostasis; stress response; stress signature
    DOI:  https://doi.org/10.1093/g3journal/jkag245
  55. Nature. 2026 Sep 14.
      Each stage of the Central Dogma contributes to proteome diversity through mechanisms such as heterozygosity, somatic mutations, transcriptional errors, and translational errors. As a result, a diverse array of protein variants can coexist within a single proteome, such as that of humans. However, until now, methods to detect, quantify, and evaluate the functional consequences of these variants have been lacking. Here we examined a large-scale proteogenomic dataset from 29 healthy human tissues and uncovered 13,910 confidently localized variants representing 7,215 unique single amino acid substitutions co-existing alongside their corresponding reference proteoforms 1.We found that the abundance of both genetic (SNP's, somatic mutations) and mistranslated protein variants mirrors their allele frequencies in the human population. Moreover, we show that non-genetic substitutions may provide a distinct route for exploring protein sequence space, circumventing the mutational constraints imposed by the genetic code. In addition, we provide experimental validation of non-genetic substitution on selected purified proteins. We demonstrate specific and recurring non-genetic variation patterns upon amino acid starvation in proteome-wide analyses of cancer-derived cell lines and identify hundreds of substituted non-genetic proteoforms that recur consistently in multiple healthy individuals or map to annotated protein functional sites. We propose that these substitutions constitute a novel class of functional protein phenotypic variants. Collectively, our findings indicate that non-genetic amino acid substitutions in human proteins provide an abundant source to expanding the functional proteome.
    DOI:  https://doi.org/10.1038/s41586-026-11124-z
  56. Mol Cell. 2026 Sep 17. pii: S1097-2765(26)00586-1. [Epub ahead of print]86(18): 3822-3839.e7
      Traditional deep mutational scanning (DMS) encodes every single amino-acid substitution from a wild-type sequence. We hypothesize that combinatorial DMS (CDMS) libraries, incorporating all mutations in all combinations, can enable the discovery of high-affinity protein (super)binders by capturing epistatic, non-linear amino-acid interactions. Here, we introduce origin-independent and context-exhaustive high-throughput integration of combinatorial DMS libraries (ORCHID), which systematically maps regions of wild-type-independent epistasis across all mutational contexts and trajectories. For benchmarking, we build a high-throughput peptide display assay measuring PIN1WW-domain affinity for a CDMS peptide library containing phosphoserine via amber codon suppression. ORCHID raises prediction accuracy by 45% over non-epistatic models. We identify and structurally characterize two epistatic superbinders, SPY-tide and LYR-tide, binding 3- to 5-fold tighter than current optimal PIN1WW-domain binders through "fold-and-turn" and register-shifted conformational changes. We also identify two molecular determinants of epistasis, PIN1F25 and PIN1R14, that natively encode non-linear binding and, when mutated, abolish it. Hence, natural proteins recognize peptides non-linearly, offering opportunities for improved binder design.
    Keywords:  CDMS; combinatorial deep mutational scanning; epistasis; molecular biology; protein function
    DOI:  https://doi.org/10.1016/j.molcel.2026.08.017
  57. Nat Commun. 2026 Sep 17. pii: 9929. [Epub ahead of print]17(1):
      Non-catalytic heterobifunctional protein binders promise to expand the range of therapeutic options by establishing complexes between key target proteins and accessory presenter proteins equipped with additional properties. Here, we systematically investigate the rational design of such molecules, explore the biochemical basis of complex formation and determine how they achieve cellular efficacy using the endogenously expressed immunophilin FKBP12 as presenter protein and the transcriptional regulator BRD4 as target protein. We present classes of bifunctional molecules that enable selective, FKBP12-dependent killing of specific cell types at subnanomolar concentrations and allow to differentiate between closely related bromodomains of the BET family. We propose that the strongly potentiated efficacy of these bifunctional compounds is based on cellular enrichment through binding to the highly abundant presenter protein FKBP12, a mechanism we term "CellTrap". Our findings substantiate the concept that highly expressed, non-essential proteins can be repurposed as selective recruiters to expand therapeutic windows of existing small-molecule inhibitors, opening new avenues for designing targeted drugs with improved cell-type specificity.
    DOI:  https://doi.org/10.1038/s41467-026-77460-w
  58. Sci Adv. 2026 Sep 18. 12(38): eaeh4755
      Human interferon-β (IFN-β), a type I IFN, is critical for effective innate immunity and is also an approved disease-modifying therapeutic. While genetic variations in several components of the type I IFN system are associated with infectious and immune diseases, consequences of IFNB1 variation remain unexplored. Here, we functionally evaluated 70 naturally occurring rare protein-altering IFNB1 variants detected in humans globally. Twenty-five percent of all protein variants were found to be substantially less active than the common reference IFN-β, a phenotype that for many correlated with retention in the ER and poor secretion, or to compromised interaction with IFNAR1/IFNAR2 cell surface receptors. Notably, three IFNB1 variants, including one found to be homozygous in a single individual, encode IFN-β proteins with enhanced capacity to engage IFNAR1/IFNAR2 and which exhibit greater signaling and antiviral potency than common IFN-β. Our mechanistic survey of natural human IFN-β functional diversity provides a framework to explore both disease associations and the optimization of human IFN-β in next-generation therapeutics.
    DOI:  https://doi.org/10.1126/sciadv.aeh4755
  59. Cancer Cell. 2026 Sep 14. pii: S1535-6108(26)00382-X. [Epub ahead of print]44(9): 1882-1892.e7
      Adult diffuse gliomas are composed of malignant cell states interwoven with the non-malignant brain microenvironment. Here, we combine spatial transcriptomics and spatial proteomics of isocitrate dehydrogenase (IDH)-mutant gliomas to define organizational principles across histological grades. In low-grade tumors, spatial organization is shaped by underlying brain anatomy. We identify a functional white-gray matter junction that restricts cortical invasion and is associated with marked changes in tumor composition and cellular phenotypes. This junction is preferentially traversed by oligodendrocyte progenitor (OPC)-like malignant cells, suggesting a role in tumor expansion. In contrast, tumors with intermediate histological features are largely disorganized, with few recurring interactions between cancer cell states and microenvironmental cell types. In high-grade tumors, hypoxia-associated structure emerges, resembling IDH-wild-type glioblastoma. Together, these findings reveal two independent axes of spatial organization-from anatomy-driven structure in low-grade tumors to hypoxia-driven organization in high-grade tumors-and establish a framework linking tumor grade to recurrent spatial interactions.
    Keywords:  IDH-mutant glioma; spatial omics; spatial organization; tumor invasion; tumor microenvironment
    DOI:  https://doi.org/10.1016/j.ccell.2026.08.005