Cell Stem Cell细胞·干细胞
Cell Stem Cell(英文缩写 CELL STEM CELL),ISSN 1934-5909,eISSN 1875-9777,中文译名:细胞·干细胞 是一本学术期刊。本页汇总该期刊的最新影响因子、分区信息以及最新收录于 PubMed 的文献,帮助您快速了解期刊全貌。
发文量统计区间：2025-09-27 至 2026-09-27，按本站收录文献的发表日期统计。
期刊介绍
历年影响因子趋势
| JCR 数据年份 | 影响因子 | JCR 分区 |
|---|---|---|
| 2021 | 25.269 | Q1 |
| 2022 | 23.900 | Q1 |
| 2023 | 19.800 | Q1 |
| 2024 | 20.400 | Q1 |
| 2025 | 23.300 | Q1 |
Cell Stem Cell 最新收录文献
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1. Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease.
PMID:日期:2026-09-24Ex vivo hematopoietic stem cell (HSC) gene therapy is effective for non-malignant blood disorders including sickle cell disease (SCD), but requires hospitalization, ex vivo cell manipulation, and conditioning. Direct in vivo gene delivery could remove these barriers and widen access. Here, we explore phagocytosis-shielded lentiviral and alpha-retroviral vectors pseudotyped with the baboon endogenous retrovirus glycoprotein variant BaEVRLess for in vivo HSC gene transfer. Vector injection into mobilized humanized mice yielded up to 8.8% gene marking in hCD45+ cells, which chemoselection enriched to 70% of hCD45+ cells and 54% of HSCs. Barcode analysis showed polyclonal reconstitution in over 90% of mice, which remained stable in secondary recipients. For SCD, we targeted BCL11A and ZNF410 selectively in erythroid cells using miRNA-embedded shRNAs to derepress gamma-globin, reaching therapeutically relevant levels of 61.5% of beta-like globins. In summary, BaEVRLess-pseudotyped lentiviral vectors enable clinically relevant in vivo gene transfer and fetal globin induction for SCD.
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2. Immunoids: Building immunocompetent organoids from human pluripotent stem cells.
PMID:日期:2026-09-24Pluripotent stem cell-derived immunocompetent organoids and assembloids, termed here "immunoids," are becoming important human models for studying immune-tissue interactions. Advances across organ systems now include incorporation of mature and immature immune populations into multiple organoid types, multilineage differentiation, and endogenous immune co-development. Defining standards for immunoid fabrication will be essential for improving physiological modeling and clinical translation. In the coming years, as these platforms mature, immunoids are poised to transform research by advancing our understanding of human development, disease mechanisms, and therapeutic responses, becoming a cornerstone of precision medicine.
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3. Genomic and transcriptomic quality control for an autologous iPSC-derived cell therapy for Parkinson's disease.
3. 用于帕金森病的自体iPSC衍生细胞疗法的基因组和转录组质量控制PMID:日期:2026-09-17Toward development of an autologous, induced pluripotent stem cell (iPSC)-based cell therapy for Parkinson's disease (PD), we demonstrate successful, reproducible genomic and transcriptomic qualification of patient-derived dopaminergic neuron precursor cells (DANPCs) across multiple donors. Our analysis includes whole-genome sequencing data from fibroblasts, iPSCs, and DANPCs and the development of NeuriTest, an RNAseq-based bioinformatic analysis of DANPCs designed to predict cell quality based on empirical animal data. Autologous cell therapies are immune matched to the patient, potentially augmenting durability of benefit compared to allogeneic cells while negating the need for immunosuppression and accompanying side effects. Patient-specific iPSCs are an autologous cell source that can be differentiated to dopaminergic neurons, the cell type lost in PD. We report here our preclinical manufacturing strategy and results demonstrating efficacy in a PD rodent model and safety in a 9-month GLP toxicology study.
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4. H3K27M drives OPC stemness and intrathecal therapeutic vulnerability in brainstem glioma organoids.
PMID:日期:2026-09-16Brainstem gliomas, particularly H3K27M-mutant diffuse midline gliomas (DMGs), lack effective therapies owing to anatomic inaccessibility, intact blood-brain barrier, and treatment resistance. Conventional models have low establishment rates and fail to preserve the native tumor microenvironment, limiting translational study for this rare disease. Here, we established a patient-derived brainstem glioma organoid (BSGO) platform with high success, faithfully recapitulating parental tumor features. Multi-omics and functional assays revealed that DMGs maintain oligodendrocyte precursor cell (OPC) stemness by dysregulating ACTIVIN signaling. To overcome delivery barriers and target stemness, we conducted an intrathecal drug screening and identified triple intrathecal therapy (methotrexate + cytarabine + dexamethasone, TIT) as a potent regimen against DMGs. In DMG patients, organoid-avatar guided personalized therapy induced sustained responses, with TIT-associated reductions in OPC markers reliably tracked by cerebrospinal fluid (CSF) proteomic profiling. Together, our study provides an organoid-based platform for mechanistic investigation and personalized intrathecal therapy screening and proposes TIT as a promising translational strategy for DMG.
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5. Clinical base editing for β-hemoglobinopathies across different genetic backgrounds.
PMID:日期:2026-09-07β-Hemoglobinopathies are monogenic disorders. We previously applied a transformer base editor (tBE) to reactivate fetal hemoglobin (HbF) expression, and five Chinese transfusion-dependent β-thalassemia (TDT) patients achieved transfusion independence. However, the applicability of tBE to sickle cell disease (SCD) and genetically different TDT populations remained unknown. Here, we show a four-patient descriptive report from three trials, including one African SCD patient and three TDT patients carrying mutations common in South and Southeast Asia. All patients achieve hematopoietic recovery, and red blood cell transfusions are discontinued in all cases. After more than 12 months of follow-up, all patients show durable editing, sustained high-level pan-cellular HbF expression, and transfusion independence. No vaso-occlusive episodes occur in the SCD patient. No off-target mutations, malignancies, or deaths are observed. These initial results support the feasibility of applying tBE to treat SCD and the reported TDT genotypes, warranting broader evaluation across diverse populations. ClinicalTrials.gov identifiers are as follows: NCT06328764, NCT06065189, and NCT06565026.
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6. Endothelialized callus organoids drive rapid regeneration of critical-size segmental long bone defects.
6. 内皮化骨痂类器官驱动临界尺寸节段性长骨缺损的快速再生PMID:日期:2026-09-03[中文摘要] 大块骨缺损仍然是一个重大的临床挑战，因为当前的治疗方法无法有效使其再生，也未能恢复功能。为重现早期骨折骨痂的细胞复杂性，我们通过将骨膜来源的骨骼祖细胞与内皮细胞共培养，工程化构建了人源内皮化骨痂类器官（hECOs）。这种共培养导致细胞自组装，形成空间有序的、类似骨痂的结构。内皮细胞促进了骨骼祖细胞扩增、细胞外基质成熟以及向肥大软骨的进展，这些都是软骨内成骨的标志。多组学分析鉴定出内皮细胞介导的再生程序激活，这些程序与骨骼成熟、基质重塑和血管生成相关。经过短暂的体外分化后，hECOs的宏观聚集体支持了免疫功能缺陷小鼠中宿主血管的快速长入和临界尺寸胫骨缺损的再生。供体来源细胞积极参与了早期再生，但在重塑过程中逐渐被替代，这与hECOs作为瞬态生物模板、引导宿主介导的骨再生相一致。
[英文摘要] Large bone defects remain a major clinical challenge, as current treatments cannot effectively regenerate them and fail to restore functionality. To recapitulate the cellular complexity of the early fracture callus, we engineered human endothelialized callus organoids (hECOs) by co-culturing periosteum-derived skeletal progenitors with endothelial cells. This co-culture resulted in cellular self-assembly, leading to spatially organized, callus-like structures. Endothelial cells promoted skeletal progenitor cell expansion, extracellular matrix maturation, and progression toward hypertrophic cartilage, hallmarks of endochondral ossification. Multi-omics analyses identified endothelial cell-mediated activation of regenerative programs associated with skeletal maturation, matrix remodeling, and angiogenesis. Following brief in vitro differentiation, macroscale aggregates of hECOs supported rapid host vascularization and regeneration of critical-size tibial defects in immunocompromised mice. Donor-derived cells actively contributed to early regeneration but were progressively replaced during remodeling, consistent with hECOs functioning as transient biological templates that guide host-mediated bone regeneration.
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7. Epi-Allele elicits compensatory expression of the non-targeted allele and prevents haploinsufficiency in dominant genetic diseases.
PMID:日期:2026-09-03Epigenetic regulation may underlie asymmetric allelic expression of many genes during development and disease pathogenesis. Allele-specific epigenetic modification could provide an efficient therapy for dominant genetic diseases due to heterozygous mutations. We developed an allele-specific epigenetic editing method ("Epi-Allele") for silencing pathogenic alleles and found surprisingly elevated expression of the non-targeted alleles, leaving total gene expression unchanged. Genome-wide screening revealed that such compensated allelic expression represents a common phenomenon, suggesting that the Epi-Allele approach could avoid the haploinsufficiency induced by current allele-specific silencing therapies. This notion was validated by allele-specific epigenetic remodeling of Myh6 and MYH7 genes in ameliorating cardiac phenotypes in a hypertrophic cardiomyopathy (HCM) mouse model and HCM patient iPSC-derived cardiomyocytes, respectively. Thus, Epi-Allele offers an allele-specific haploinsufficiency-free therapeutic approach for treating dominant genetic diseases.
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8. Feeling the tension: Engineered stem cells convert tumor stiffness into a drug target.
PMID:日期:2026-09-03Radiopharmaceutical theranostics remains constrained by tumors lacking a validated molecular target. Yang et al. engineer mesenchymal stem cells that sense tumor stiffness and respond by manufacturing a synthetic target in situ, making PET imaging and radionuclide therapy possible with existing clinical radioligands in tumors that were previously untargetable.
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9. Beyond compensation: Organized pathological hematopoiesis in the myelofibrotic spleen.
PMID:日期:2026-09-03Two studies in this issue of Cell Stem Cell show that splenic extramedullary hematopoiesis in myelofibrosis develops within a remodeled hematopoietic, immune, and stromal environment. Austin et al. define its cellular states, whereas Dugué et al. reveal the spatial and temporal remodeling of the stromal niche.
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10. APOE4 astrocytes call the shots on neuronal α-synuclein pathology.
PMID:日期:2026-09-03In this issue of Cell Stem Cell, Mesentier-Louro et al. use a multi-cellular integrated brain (miBrain) system to uncover mechanisms underlying the accumulation of neuronal α-synuclein (α-Syn) inclusions in APOE4 carriers. Their results suggest that APOE4 increases cholesterol levels and impairs lysosomal function in astrocytes, which release pathogenic α-Syn.