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Activins and BMPs: The yin and yang of diffuse midline gliomas 激活素与bmp：弥漫性中线胶质瘤的阴阳关系
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-10-01 DOI: 10.1016/j.stem.2026.09.002
Kavya Prasad,Oren J. Becher
{"title":"Activins and BMPs: The yin and yang of diffuse midline gliomas","authors":"Kavya Prasad,Oren J. Becher","doi":"10.1016/j.stem.2026.09.002","DOIUrl":"https://doi.org/10.1016/j.stem.2026.09.002","url":null,"abstract":"Patient-derived models of diffuse midline gliomas (DMGs) remain challenging for interrogating tumor biology and investigating novel therapeutics. Xie et al. develop a robust patient-derived brainstem glioma organoid (BSGO) platform, reveal an Activin B-ACVR2A signaling pathway that promotes DMG growth, and identify a promising triple intrathecal therapy to inhibit it.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"1 1","pages":"1573-1574"},"PeriodicalIF":23.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149090144","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Silencing the mutant allele without compromising gene dosage 沉默突变等位基因而不影响基因剂量
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-10-01 DOI: 10.1016/j.stem.2026.08.011
Lu Tian,Junhan Chen,Yinqing Li
{"title":"Silencing the mutant allele without compromising gene dosage","authors":"Lu Tian,Junhan Chen,Yinqing Li","doi":"10.1016/j.stem.2026.08.011","DOIUrl":"https://doi.org/10.1016/j.stem.2026.08.011","url":null,"abstract":"Allele-selective silencing offers a strategy to suppress pathogenic alleles in dominant genetic disorders but may alter total gene dosage when the remaining allele does not compensate. Wang et al.1 show that SNP-guided epigenetic repression of mutant alleles can elicit compensatory expression from non-targeted alleles, highlighting considerations for future therapeutic applications.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"222 1","pages":"1569-1570"},"PeriodicalIF":23.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149090143","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells 就地编辑细胞：针对造血干细胞的体内基因编辑
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-10-01 DOI: 10.1016/j.stem.2026.08.015
Sijin Luozhong,Angelica Varesi,Bowen Li
{"title":"Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells","authors":"Sijin Luozhong,Angelica Varesi,Bowen Li","doi":"10.1016/j.stem.2026.08.015","DOIUrl":"https://doi.org/10.1016/j.stem.2026.08.015","url":null,"abstract":"Traditional ex vivo hematopoietic stem cell (HSC) gene therapy faces manufacturing challenges. A recent study1 in Nature Biomedical Engineering demonstrates that antibody-engineered lipid nanoparticles achieve efficient and durable in vivo editing of HSCs in humanized mice, offering a promising therapeutic strategy for genetic blood diseases.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"26 1","pages":"1578-1579"},"PeriodicalIF":23.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149090145","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Right size, right time: The Hippo-IGF2 axis calibrates liver growth and regeneration 合适的大小，合适的时间：希波- igf2轴校准肝脏生长和再生
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-10-01 DOI: 10.1016/j.stem.2026.08.013
Wolfram Goessling
{"title":"Right size, right time: The Hippo-IGF2 axis calibrates liver growth and regeneration","authors":"Wolfram Goessling","doi":"10.1016/j.stem.2026.08.013","DOIUrl":"https://doi.org/10.1016/j.stem.2026.08.013","url":null,"abstract":"The imprinted gene IGF2 is a well-characterized growth factor regulating organ proliferation during embryogenesis, while Hippo signaling has been demonstrated as an important regulator of organ size. This study by Zhong et al.1 mechanistically connects Hippo-controlled Igf2 expression in postnatal liver growth and regeneration.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"4 1","pages":"1571-1572"},"PeriodicalIF":23.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149090141","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS 药物的制造过程是：aso递送的siRNA进入人体中枢神经系统治疗ALS
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-10-01 DOI: 10.1016/j.stem.2026.09.004
Wenxuan Guo,Justin K. Ichida
{"title":"The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS","authors":"Wenxuan Guo,Justin K. Ichida","doi":"10.1016/j.stem.2026.09.004","DOIUrl":"https://doi.org/10.1016/j.stem.2026.09.004","url":null,"abstract":"In a recent article in Nature Medicine, Chen et al. describe a first-in-human study using an ASO tag to deliver a SOD1 siRNA to the central nervous system (CNS), potentially achieving faster, more extensive, and better-tolerated SOD1-lowering in ALS patients.1","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"3 1","pages":"1580-1581"},"PeriodicalIF":23.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149090146","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Flipping the fetal switch across β-hemoglobinopathies 在β-血红蛋白病中翻转胎儿开关
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-10-01 DOI: 10.1016/j.stem.2026.09.003
Nishi Harwani,Akshay Sharma
{"title":"Flipping the fetal switch across β-hemoglobinopathies","authors":"Nishi Harwani,Akshay Sharma","doi":"10.1016/j.stem.2026.09.003","DOIUrl":"https://doi.org/10.1016/j.stem.2026.09.003","url":null,"abstract":"Liu et al. extend the application of transformer base editing of HBG1/2 promoters from transfusion-dependent β-thalassemia alone to one patient with sickle cell disease and three patients with geographically diverse β-thalassemia genotypes.1 The responses are meaningful, but scale, genomic surveillance, burden of cure, and access now define the translational test.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"26 1","pages":"1575-1577"},"PeriodicalIF":23.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149090142","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Immunoids: Building immunocompetent organoids from human pluripotent stem cells 免疫类：利用人多能干细胞构建具有免疫能力的类器官
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-09-24 DOI: 10.1016/j.stem.2026.08.016
Colin O’Hern,Zachary Miller,Mia Dionise,Satyajit Hari Kulkarni,Christopher H. Contag,Aitor Aguirre
{"title":"Immunoids: Building immunocompetent organoids from human pluripotent stem cells","authors":"Colin O’Hern,Zachary Miller,Mia Dionise,Satyajit Hari Kulkarni,Christopher H. Contag,Aitor Aguirre","doi":"10.1016/j.stem.2026.08.016","DOIUrl":"https://doi.org/10.1016/j.stem.2026.08.016","url":null,"abstract":"Pluripotent 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.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"6 1","pages":""},"PeriodicalIF":23.9,"publicationDate":"2026-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149027572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease 吞噬屏蔽逆转录病毒载体的全身递送使体内HSC基因治疗镰状细胞病成为可能
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-09-24 DOI: 10.1016/j.stem.2026.09.001
Denise Klatt,Adele Mucci,Boya Liu,Marie Peschers,Giulia Schiroli,Axel Schambach,John Manis,Myriam Armant,David T. Scadden,Els Verhoeyen,Danilo Pellin,David A. Williams,Christian Brendel
{"title":"Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease","authors":"Denise Klatt,Adele Mucci,Boya Liu,Marie Peschers,Giulia Schiroli,Axel Schambach,John Manis,Myriam Armant,David T. Scadden,Els Verhoeyen,Danilo Pellin,David A. Williams,Christian Brendel","doi":"10.1016/j.stem.2026.09.001","DOIUrl":"https://doi.org/10.1016/j.stem.2026.09.001","url":null,"abstract":"Ex 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.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"30 1","pages":""},"PeriodicalIF":23.9,"publicationDate":"2026-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149027573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Genomic and transcriptomic quality control for an autologous iPSC-derived cell therapy for Parkinson’s disease 自体ipsc衍生细胞治疗帕金森病的基因组和转录组质量控制
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-09-17 DOI: 10.1016/j.stem.2026.08.014
Andres M. Bratt-Leal,Thorsten Gorba,Roy M. Williams,Jim A. Mossman,Rachel Hills,Ha Tran,Derren Barken,Genevieve Beauvais,Daniel Fremgen,Ai Zhang,Cullen Pivaroff,Candace Lynch,Yanzheng Liu,Chandnee Chandrasekaran,David Tastad,Charlotte Bridge,Dustin R. Wakeman,Uyen Nguyen,Brent Chamberlain,Emma L. Lane,Mariah J. Lelos,Jeanne F. Loring,Edward D. Wirth III,Xiaokui Zhang
{"title":"Genomic and transcriptomic quality control for an autologous iPSC-derived cell therapy for Parkinson’s disease","authors":"Andres M. Bratt-Leal,Thorsten Gorba,Roy M. Williams,Jim A. Mossman,Rachel Hills,Ha Tran,Derren Barken,Genevieve Beauvais,Daniel Fremgen,Ai Zhang,Cullen Pivaroff,Candace Lynch,Yanzheng Liu,Chandnee Chandrasekaran,David Tastad,Charlotte Bridge,Dustin R. Wakeman,Uyen Nguyen,Brent Chamberlain,Emma L. Lane,Mariah J. Lelos,Jeanne F. Loring,Edward D. Wirth III,Xiaokui Zhang","doi":"10.1016/j.stem.2026.08.014","DOIUrl":"https://doi.org/10.1016/j.stem.2026.08.014","url":null,"abstract":"Toward 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.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"20 1","pages":""},"PeriodicalIF":23.9,"publicationDate":"2026-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148980628","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
H3K27M drives OPC stemness and intrathecal therapeutic vulnerability in brainstem glioma organoids H3K27M驱动脑干胶质瘤类器官的OPC干性和鞘内治疗易感性
IF 23.9 1区 医学
Cell stem cell Pub Date : 2026-09-16 DOI: 10.1016/j.stem.2026.08.012
Luyang Xie,Zhuang Jiang,Hang Zhou,Xiaoxue Zhu,Yibo Geng,Mingxin Zhang,Tengxian Guo,Ge Shen,Xiaoyan Li,Ligong Chen,Gairong Zhang,Changwen Xue,Tuozhang Li,Xixi Zheng,Yaqi Wang,Haitao Fu,Yuxuan Deng,Jingfeng Li,Kai Jiang,Wenhao Wu,Peng Zhang,Changcun Pan,Shaobo Shan,Yujie Tang,Huan Li,Liang Wang,Zhen Wu,Cheng Xu,Liwei Zhang
{"title":"H3K27M drives OPC stemness and intrathecal therapeutic vulnerability in brainstem glioma organoids","authors":"Luyang Xie,Zhuang Jiang,Hang Zhou,Xiaoxue Zhu,Yibo Geng,Mingxin Zhang,Tengxian Guo,Ge Shen,Xiaoyan Li,Ligong Chen,Gairong Zhang,Changwen Xue,Tuozhang Li,Xixi Zheng,Yaqi Wang,Haitao Fu,Yuxuan Deng,Jingfeng Li,Kai Jiang,Wenhao Wu,Peng Zhang,Changcun Pan,Shaobo Shan,Yujie Tang,Huan Li,Liang Wang,Zhen Wu,Cheng Xu,Liwei Zhang","doi":"10.1016/j.stem.2026.08.012","DOIUrl":"https://doi.org/10.1016/j.stem.2026.08.012","url":null,"abstract":"Brainstem 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.","PeriodicalId":9665,"journal":{"name":"Cell stem cell","volume":"74 1","pages":""},"PeriodicalIF":23.9,"publicationDate":"2026-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148973298","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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