bims-scepro Biomed News
on Stem cell proteostasis
Issue of 2026–07–12
sixteen papers selected by
William Grey, University of York



  1. J Clin Transl Res. 2025 Oct 29. 11(5): 50-68
      Background. Hematopoietic stem cells (HSCs) reside in the bone marrow and are responsible for the life-long production of blood cells by balancing quiescence, self-renewal, and differentiation. A major feature distinguishing quiescent HSCs from their activated counterparts is a shift in the metabolic profile including changes in glycolytic flux and mitochondrial oxidative metabolism. Disruptions to HSC homeostasis can lead to hematologic diseases such as bone marrow failure or clonal hematopoiesis and even oncogenic transformation to form leukemic stem cells (LSCs). Like that of HSCs, LSCs retain stem-like characteristics but also gain features of malignancy including drug resistance and a hijacked metabolism that exhibit distinct metabolic profiles that can underlie their pathogenesis. The aim of this review is to summarize the key metabolic characteristics that distinguish healthy quiescent and active HSCs as well as oncogenic LSCs. Here we also explore the modern tools used to investigate the metabolome and how they can reveal novel metabolites, metabolic interactions and pathways, and targets for diagnosis or therapeutic intervention of hematologic diseases. Understanding and interrogating changes to the metabolic profiles of healthy and leukemic stem cells may lead to the development of innovative techniques, technologies, and therapeutics. In turn, these advances can be used for the identification, treatment, and prevention of hematologic disease. By better understanding their metabolome, therapies can be designed to target the unique metabolic pathways, dependencies, and resistance mechanisms of LSCs.
    Keywords:  hematopoietic stem cells; leukemic stem cells; metabolism; metabolomics
    DOI:  https://doi.org/10.36922/jctr025320053
  2. Tissue Eng Part C Methods. 2026 Jul 10. 19373384261463760
      Hematopoietic stem and progenitor cells (HSPCs) are precisely organized within specialized bone marrow niches containing hematopoietic and nonhematopoietic cell types. Physiologically accurate human models of this niche environment that include functional HSPCs have been lacking until recently. Using induced pluripotent stem cells (iPSCs) grown in mixed-matrix hydrogels, vascularized three-dimensional human bone marrow organoids (hBMOs) can now be generated that contain mesenchymal, endothelial, and hematopoietic cells, all vital components of the bone marrow niche. While hBMOs have been shown to support engraftment of normal and malignant cells, there are numerous opportunities to use hBMOs for developing a wider experimental toolkit to test gene functions and stress responses. Here, we establish two new applications for the hBMO platform. First, we demonstrate successful engraftment of gene-edited CD34+ cells from healthy donors, enabling direct investigation of gene-specific effects on human hematopoiesis in a defined microenvironment. Gene-edited engrafted HSPCs are maintained in hBMOs for 7 days and undergo multilineage differentiation. Second, we adapted a method to induce stress erythropoiesis-a response to acute anemia in mice and humans-in hBMOs, resulting in robust expansion of immunophenotypically defined hematopoietic progenitors and erythroid populations. These two advances expand the laboratory uses for hBMOs and establish this system as a versatile platform for studying human hematopoiesis and erythropoiesis, stress responses, and gene functions within a physiologically relevant bone marrow microenvironment.
    Keywords:  hematopoiesis, bone marrow organoid, gene editing, engraftment
    DOI:  https://doi.org/10.1177/19373384261463760
  3. Nat Commun. 2026 Jul 06.
      The stem cell niche is a specialised microenvironment essential for maintaining haematopoietic stem cells (HSCs). Here we identify a distinct subset of mesenchymal stem cells (MSCs) expressing integrin α8 (Itga8⁺ MSCs) within the bone marrow (BM) endosteum. These cells exhibit distinct MSC properties and higher haematopoietic supportive activity than other MSC subsets. Depletion of Itga8⁺ MSCs decreased HSC numbers, reduced quiescence of endosteal HSCs and diminished BM repopulating capacity. Itga8⁺ MSCs support haematopoiesis through cell adhesion-related mechanisms. Single-cell RNA sequencing further identified Itga8⁺ MSCs as a distinct subpopulation within bone-lining cells. Moreover, we identified Mfap4 as a candidate factor expressed in Itga8⁺ MSCs, and Mfap4 supported the BM reconstitution capacity of HSCs. These results suggest that Itga8⁺ MSCs represent a distinct niche cell population for HSC maintenance. This work provides new insights into HSC regulation and strategies to optimise in vitro HSC maintenance and enhance in vivo engraftment.
    DOI:  https://doi.org/10.1038/s41467-026-75276-2
  4. J Proteome Res. 2026 Jul 10.
      Recent advances in mass spectrometry-based single-cell proteomics (SCP) technologies have revolutionized the SCP field. However, current SCP approaches generally employ sub-μL to 1 μL processing volumes for effective single-cell sample preparation using either ultralow-volume specialized devices or a 384-well plate by frequently adding water to compensate for evaporation, which limits their broad accessibility and analytical robustness. Here, we report a robust and convenient SCP method termed iSOP (improved surfactant-assisted one-pot processing) for the processing of single cells at a low μL processing volume using a 384-well plate with tight sealing to avoid sample drying loss. After systematic optimization, 3 μL was selected for iSOP as the processing volume, with a mixture of trypsin and Lys-C enzymes (2 ng of each enzyme) in terms of robustness, sensitivity, and operation convenience. With a commonly accessible LC-MS platform, iSOP-MS can detect and quantify ∼1200-1800 protein groups from single HeLa or MCF7 cells. Application of iSOP-MS to two neuroblastoma cell lines enabled reliable identification of an average of ∼1700 and ∼2050 protein groups from single BE2-C and SK-N-SH cells, respectively, and precise characterization of cellular heterogeneity. When compared to other available SCP methods, iSOP-MS is more robust and convenient for routine, cost-effective, quantitative SCP analysis.
    Keywords:  384-well plate; cellular heterogeneity; iSOP-MS; low μL processing volume; single-cell proteomics (SCP)
    DOI:  https://doi.org/10.1021/acs.jproteome.6c00063
  5. Signal Transduct Target Ther. 2026 Jul 07. pii: 262. [Epub ahead of print]11(1):
      Multiple myeloma (MM) is characterized by the production and secretion of large quantities of immunoglobulins, making this malignancy highly dependent on mechanisms that maintain cellular proteostasis. While significant clinical progress has been made by targeting the degradative branch of proteostasis, much less attention has been given to the biosynthetic branch. In this study, we demonstrated that inhibiting COPII-dependent endoplasmic reticulum (ER) export induces cell death in several MM cell lines and primary patient-derived cells. The induction of cell death was dependent on the secretory status of MM cells. Blocking ER export in secretory MM cells caused the accumulation of misfolded proteins, which activated ER-associated degradation (ERAD). Consequently, we observed an ERAD-dependent increase in the levels of free cytosolic amino acids and a subsequent activation of mTORC1 signaling. Simultaneously, we observed mitochondrial dysfunction. These alterations resulted in a mismatch between the increased energy demand due to mTORC1 activation, and the disrupted energy supply from mitochondrial impairment. This energetic imbalance results in homeostatic collapse and cell death of secretory MM cells. The therapeutic potential of the concept was demonstrated in two in vivo myeloma models. These findings suggest that the ER export machinery could be a promising therapeutic target in multiple myeloma.
    DOI:  https://doi.org/10.1038/s41392-026-02833-y
  6. Nature. 2026 Jul 08.
      The short-term and long-term effects of genotoxic pre-transplant conditioning remain barriers to the broader application of haematopoietic stem/progenitor cell (HSPC) transplantation and gene therapies1-4. Although monoclonal antibodies targeting KIT have been proposed as alternatives to chemotherapy or radiotherapy5-7, their pharmacokinetics hinder clinical applications owing to the risk of depleting transplanted HSPCs. Here, to address this issue, we identified amino acid changes in the extracellular domain of KIT that disrupt the binding of two therapeutic monoclonal antibodies8,9, which impair stem cell factor (SCF)-mediated signalling without affecting KIT expression or functionality. We exploited adenine base editing10 or prime editing11 to efficiently introduce these mutations in HSPCs and combined them with the disruption of the BCL11A erythroid enhancer to promote expression of fetal haemoglobin (HbF)12,13, a therapeutic approach for several haemoglobinopathies. This strategy enables in vivo co-selection of gene-engineered cells to reach the threshold required to provide therapeutic benefit in patients affected by sickle cell disease and β-thalassaemia. We show progressive enrichment of KIT plus BCL11A multiplex-edited haematopoiesis under selective pressure with KIT monoclonal antibody, in vitro and in vivo. We report that extended treatment with anti-KIT regimens leads to superior in vivo enrichment while avoiding clonal selection, as assessed by a lentiviral barcoded library. Finally, by overcoming the limitations of monoclonal antibody pharmacokinetics, epitope editing enables novel haematopoietic replacement regimens that are not limited by on-target graft elimination, allowing prolonged immune-based conditioning that maximizes haematopoietic niche clearance without chemo-radiotherapy or monoclonal antibody wash-out.
    DOI:  https://doi.org/10.1038/s41586-026-10737-8
  7. Nat Commun. 2026 Jul 10.
      Chromosome copy number variations are poorly understood drivers of human malignancies. -7/del(7q) is common in acute myeloid leukemia, confers a poor prognosis, and is thought to harbor several tumor suppressors. Previously, we identified the histone methyltransferase KMT2C as a tumor suppressor in this region. Here, through a differentiation CRISPR screen in hematopoietic stem and progenitor cells, we find that the mitochondrial iron transporter ABCB8 is essential for their differentiation. ABCB8 deficiency accelerates leukemogenesis in vivo and disrupts iron homeostasis, reducing cytoplasmic iron availability and impairing iron-dependent enzymes, including the histone demethylase KDM6A. Consequently, ABCB8 loss elevates H3K27me3 levels, repressing differentiation genes in an iron- and KDM6A-dependent manner. Notably, ABCB8 and KMT2C, neighboring genes on 7q, cooperatively regulate H3K27me3 to suppress leukemogenesis. Our findings reveal ABCB8 as a tumor suppressor in -7/del(7q) acute myeloid leukemia and uncover an epigenetic collaboration between neighboring tumor suppressors, driven by iron-mediated chromatin remodeling.
    DOI:  https://doi.org/10.1038/s41467-026-75292-2
  8. Sci Transl Med. 2026 Jul 08. 18(857): eadx3847
      Chemotherapy resistance in acute myeloid leukemia (AML) remains a major clinical challenge. Integration of multiomic profiling and in vivo functional genomics revealed splicing dysregulation as a determinant of chemoresistance in AML. We uncovered a network involving the splicing regulator SRRM1 and the CLK1/4 and PAK1 kinase families as vulnerabilities in chemoresistant AML cells. Both kinase families are hyperactivated in chemoresistant cells, promoting SRRM1 phosphorylation and altering its scaffolding function. We also identified a relapse-associated PAK1 variant, c.1429G>T p.(Ala477→Ser), that confers chemotherapy resistance. Combined PAK1 and CLK1/4 inhibition recapitulated the splicing changes induced by SRRM1 loss, preferentially targeting chemoresistant AML and enhancing chemotherapy efficacy in cell lines, primary cells, and mouse models. Last, we pinpointed MAP2K5 as a critical downstream effector because missplicing of exons 17 and 18 of MAP2K5 upon SRRM1 depletion sensitized cells to chemotherapy. Our findings highlight a therapeutic strategy to overcome AML relapse by targeting splicing dysregulation.
    DOI:  https://doi.org/10.1126/scitranslmed.adx3847
  9. Stem Cell Rev Rep. 2026 Jul 09.
      Stem cell fate decisions-whether to self-renew, differentiate, or senesce-are inextricably linked to the metabolic identity and quality-control status of mitochondria. The ubiquitin-proteasome system and selective autophagy pathways assemble into an integrated surveillance network at the mitochondrial outer membrane that gauges organelle health, sculpts morphology, and transduces metabolic information into lineage-determining transcriptional programmes. This Review examines how the ubiquitination machinery-spanning the canonical PINK1-Parkin axis and non-Parkin E3 ligases including MARCH5, MUL1, and the emerging Cullin-RING component RBX2-orchestrates outer-membrane protein degradation, mitochondria-derived vesicle biogenesis, and the balance between fusion and fission. We discuss how these post-translational events govern stem cell identity across haematopoietic, muscle, neural, mesenchymal, and pluripotent compartments. Recent 2024-2025 advances include an Nicotinamide Adenine Dinucleotide (NAD+)-dependent metabolic checkpoint governing haematopoietic stem cell activation and aging, the crystallographic resolution of USP30 inhibitor binding, molecular glue activators that allosterically enhance Parkin RING-domain activity, ClpP-based mitochondria-targeted PROTAC platforms, and HIF-1α/BNIP3-mediated pharmacological rejuvenation of aged mesenchymal stem cells. We further discuss the WAC-PINK1-Parkin axis in mesenchymal stem cell aging, the bidirectional interplay between reactive oxygen species and E3 ligase activity, and the ACC1-FIS1 ubiquitination axis. Finally, we consider the cell-type-specific calibration of mitochondrial ubiquitination as a unifying principle for precision therapeutics and the inverted quality-control logic exploited by cancer stem cells. We propose that the cell-type-specific calibration of mitochondrial ubiquitination-whereby identical molecular events carry divergent functional consequences across stem cell compartments-offers a unifying framework for precision therapeutics.
    Keywords:  Mitochondrial dynamics; Mitochondrial ubiquitination; Mitophagy; PINK1-Parkin; Stem cell fate
    DOI:  https://doi.org/10.1007/s12015-026-11189-3
  10. Science. 2026 Jul 09. 393(6807): 188-194
      Lysine acetyltransferases (KATs) cooperate with oncogenes such as c-Myc, estrogen receptor, and lysine methyltransferase 2A (KMT2A) fusions to sustain malignant programs. Targeting of KAT proteins has shown clinical efficacy; however, achieving homolog selectivity for most KATs remains a major challenge. By extending cereblon (CRBN)-based molecular glues beyond the canonical degron space, we developed an exquisitely selective degrader of KAT2A. Cryo-electron microscopy revealed that CRBN recruits KAT2A independently of a degron; instead, the molecular glue engages a surface-exposed tyrosine, mimicking antibody-like molecular recognition. Selective KAT2A degradation leads to potent ablation of histone H3 lysine 9 acetylation (H3K9Ac), antiproliferative effects in acute myeloid leukemia cell lines, and in vivo efficacy in a patient-derived xenograft model, establishing KAT2A as a targetable vulnerability to treat a wide range of malignancies. More generally, degron-independent recruitment extends the CRBN-targetable proteome.
    DOI:  https://doi.org/10.1126/science.aef5391
  11. Cancer Cell. 2026 Jul 09. pii: S1535-6108(26)00295-3. [Epub ahead of print]
      Menin inhibition, an approved therapy for KMT2A-rearranged and NPM1 mutant acute leukemia, is accompanied by decreased platelet counts in 15-20% of heavily pre-treated patients. While studying the mechanism underlying this effect, we discovered that menin inhibition reduced the numbers of megakaryocyte progenitors in human CD34+ cultures and in mice. Because megakaryocytes are key drivers of myeloproliferative neoplasms (MPNs), we investigated the extent to which menin inhibition ameliorates MPN phenotypes. We found that the menin inhibitor revumenib has potent anti-tumor activity, synergizes with ruxolitinib, and shows only subtle effects on healthy mice. Moreover, revumenib suppressed megakaryopoiesis of primary MPN patient specimens in vitro and in vivo. Importantly, genetic knockout of MEN1 and its target MEF2C phenocopied the action of revumenib, confirming an on-target effect of the drug. Together, we reveal menin as a dependency in proliferative megakaryocytes and support further evaluation of menin inhibition as a potential therapy for MPNs.
    Keywords:  megakaryocyte; menin; myeloproliferative neoplasms; revumenib
    DOI:  https://doi.org/10.1016/j.ccell.2026.06.008
  12. Blood. 2026 Jul 07. pii: blood.2025032630. [Epub ahead of print]
      Multiple myeloma progresses from precursor states to active disease, and studying tumor microenvironment (TME) evolution across these stages is key to understanding immune dysregulation and therapeutic resistance. Here, we integrated paired single-cell RNA, T-, and B-cell receptor sequencing data from bone marrow samples of 235 patients spanning the disease spectrum. This dataset revealed dynamic changes in the abundance and functional states of diverse immune populations, including T-, natural killer, B-, and myeloid cells. Using non-negative factorization of cell-subset composition, we identified five reproducible TME subtypes, or "ecotypes," defined by coordinated cellular architectures. These ecotypes captured structured variation beyond disease stage and were associated with distinct cell-cell communication networks, cytokine signaling landscapes, transcription factor programs, and shared gene modules, reflecting coordinated immune adaptation to microenvironmental constraints. By linking tumor features with immune changes and ecotype distributions, we identified context-dependent associations influenced by both disease biology and treatment effects on the TME. We found that an ecotype enriched for bone marrow-resident populations and limited immune infiltration was associated with tumor expansion and inferior clinical outcomes, whereas ecotypes reflecting T-cell functional states showed distinct associations with immunotherapy response and survival. Our study provides a comprehensive single-cell immune atlas of multiple myeloma and its precursors, offering insights into TME organization and progression, and may guide development of stage-specific or ecotype-targeted immunotherapies.
    DOI:  https://doi.org/10.1182/blood.2025032630
  13. Protein Sci. 2026 Aug;35(8): e70703
      Mitochondria respond to proteotoxic stress through the mitochondrial unfolded protein response, traditionally viewed as a transcriptional program that restores proteostasis by inducing chaperones and proteases. Emerging evidence indicates that mitochondrial membrane remodeling constitutes an additional adaptive component of this response. Regulated changes in mitochondrial lipid composition, particularly involving the signature phospholipid cardiolipin, support mitochondrial function during stress by stabilizing protein import machineries, promoting mitochondrial protein biogenesis, and facilitating recovery from dysfunction. In addition, stress originating in other organelles, especially the endoplasmic reticulum, reshapes mitochondrial membranes through altered lipid biosynthesis, inter-organelle lipid trafficking, and stress signaling pathways. These findings suggest that mitochondrial membrane remodeling represents a regulatory layer of organelle quality control integrated within interconnected stress response networks and may provide new opportunities to enhance mitochondrial resilience in disease.
    Keywords:  ER–mitochondria crosstalk; cardiolipin; mitochondrial membrane remodeling; mitochondrial protein biogenesis; mitochondrial unfolded protein response (UPRmt); organelle stress signaling
    DOI:  https://doi.org/10.1002/pro.70703
  14. Nature. 2026 Jul 08.
      Acute myeloid leukaemia (AML) is an aggressive blood cancer characterized by the unregulated proliferation of immature myeloblasts. Gene mutations have been shown to have a large effect on pathogenesis, inter-tumour heterogeneity and clinical outcomes in AML1-8; however, the role of epigenetic alterations in these respects has been investigated less extensively. Here we use ATAC-seq (assay for transposase-accessible chromatin with sequencing) in a cohort of 1,563 individuals with a recent diagnosis of AML (the 'eCHROMA' cohort) to show that AML can be classified into 16 subgroups on the basis of chromatin accessibility profiles. Multiomics analyses of gene mutations, the transcriptome, DNA methylation and histone marks show that these ATAC subgroups exhibit distinct driver mutations, differentiation states, gene expression, DNA methylation and super-enhancer profiles, and are also associated with clinical outcomes. These findings were validated in independent cohorts. Single-cell ATAC sequencing reveals that all leukaemic cells in each subgroup share a common chromatin accessibility profile, which suggests that subgroup-specific epigenomic fingerprints underlie the ATAC-based classification. Mechanistically, the subgroups have distinct gene-regulatory networks that are driven by the activities of key transcription factors in haematopoiesis, and in which subgroup-specific super-enhancers have a pivotal role. Multiomics single-cell analysis further reveals deregulated trajectories of differentiation coupled with chromatin accessibility and gene expression. Notably, ATAC subgroups have an independent prognostic effect, compared with genomic classification, and are associated with particular drug sensitivities. In summary, ATAC-based chromatin profiling, combined with multiomics data, provides insights into AML pathogenesis beyond genomics and constitutes a valuable resource for AML research.
    DOI:  https://doi.org/10.1038/s41586-026-10703-4
  15. Res Sq. 2026 Jul 02. pii: rs.3.rs-9916651. [Epub ahead of print]
      The polarized organization of cellular constituents is vital for cell migration, fate decisions, and tissue organization and often altered in disease. However, its quantification remains challenging because cells vary in size, shape, marker expression, and change over time. As existing approaches lack throughput, discard spatial information, and cannot measure polarity dynamics, we introduce CellPolariS, a novel image analysis framework to quantify polarity in diverse cell types from different tissues and in living cells. Unlike other methods, CellPolariS uses spatial subcellular organization to quantify the number, direction, magnitude, and concentration of cellular structures. We identify that differences in cell morphology and marker expression between cells are critical confounding factors that impair polarity quantifications. Through extensive validation and quantification of diverse cell types, including T-cells, natural killer cells, zygotes, neurons, yeast, and bacteria, we demonstrate that CellPolariS corrects for these effects and provides precise measurements of the polarization of Tubulin, Actin, and other structures. In addition, we analyze hundreds of thousands of primary stem and progenitor cells, map how Tubulin, CDC42, and Actin polarity change throughout hematopoietic differentiation, and discover that polarity increases during erythroid differentiation. Using long-term imaging of living cells, we further show that polarized subcellular structures are highly dynamic and can quickly change from polar to non-polar states, suggesting that cell polarity is more dynamic than previously appreciated.
    DOI:  https://doi.org/10.21203/rs.3.rs-9916651/v1
  16. bioRxiv. 2026 Jun 29. pii: 2026.06.26.734844. [Epub ahead of print]
      Hematopoietic stem cells are required to regenerate the blood system throughout life. We previously discovered that transitions between quiescent stem and multipotent progenitor states are controlled by mutual inhibition between Mecom and Cdk6 , with upstream regulation from the insulin-like growth factor (IGF) signaling pathway. To investigate the dynamics of exit from quiescence and the stem-to-multipotent cell state transition, we modeled the Mecom - Cdk6 regulatory network via coupled nonlinear differential equations. Bifurcation analysis revealed that the model permits tetrastability, with two stable intermediate states, suggesting that stem cell exit from quiescence proceeds via multiple fine-scale transitions. Perturbation of Mecom self-activation reorganized the multistable landscape, producing two IGF-dependent landscapes with distinct geometries. At high IGF, transitions proceeded only through an intermediate state, whereas at low IGF, a distinct landscape emerged permitting direct transitions between cell states. Stochastic simulations and minimum action path analysis showed that the multipotent attractor is deep at high IGF, whereas low IGF promotes a transition to quiescence by stabilizing the stem cell state. Simulated pharmacological intervention via CDK4/6 inhibitors destabilized the multipotent state and favored transitions towards a more stem-like quiescent state. Together, these results demonstrate how IGF signaling, Mecom self-activation, and Cdk6 inhibition jointly shape early stem cell fate decisions by dictating the accessible cell states on multistable landscapes and the transition paths that connect them.
    DOI:  https://doi.org/10.64898/2026.06.26.734844