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generated hematopoietic stem cells from genome edited induced pluripotent stem cells are functional in platelet-targeted gene therapy of murine hemophilia A.

作者信息

Wang Dawei, Zhang Guowei, Gu Junjie, Shao Xiaohu, Dai Yuting, Li Jianfeng, Pan Xiaohong, Yao Shuxian, Xu Aining, Jin Ying, Huang Jinyan, Shi Qizhen, Zhu Jiang, Xi Xiaodong, Chen Zhu, Chen Saijuan

机构信息

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.

National Research Center for Translational Medicine, Shanghai Jiao Tong University School of Medicine, Shanghai, China.

出版信息

Haematologica. 2020 Apr;105(4):e175-e179. doi: 10.3324/haematol.2019.219089. Epub 2019 Jul 11.

DOI:10.3324/haematol.2019.219089
PMID:31296582
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7109733/
Abstract
摘要

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Stem Cells in the Treatment of Disease.干细胞在疾病治疗中的应用
N Engl J Med. 2019 May 2;380(18):1748-1760. doi: 10.1056/NEJMra1716145.
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Respecifying human iPSC-derived blood cells into highly engraftable hematopoietic stem and progenitor cells with a single factor.用单一因子将人诱导多能干细胞衍生的血细胞重编程为具有高植入能力的造血干细胞和祖细胞。
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Haematopoietic stem and progenitor cells from human pluripotent stem cells.源自人类多能干细胞的造血干细胞和祖细胞。
Nature. 2017 May 25;545(7655):432-438. doi: 10.1038/nature22370. Epub 2017 May 17.
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Induced Pluripotent Stem Cells Meet Genome Editing.诱导多能干细胞与基因组编辑相遇。
Cell Stem Cell. 2016 May 5;18(5):573-86. doi: 10.1016/j.stem.2016.04.013.
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Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting.人类重症联合免疫缺陷的建模及通过CRISPR/Cas9增强基因靶向进行的纠正
Cell Rep. 2015 Sep 8;12(10):1668-77. doi: 10.1016/j.celrep.2015.08.013. Epub 2015 Aug 28.
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Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.利用 CRISPR/Cas9 介导的种系 DNA 编辑预防小鼠肌肉萎缩症。
Science. 2014 Sep 5;345(6201):1184-1188. doi: 10.1126/science.1254445. Epub 2014 Aug 14.
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Development and applications of CRISPR-Cas9 for genome engineering.用于基因组工程的CRISPR-Cas9技术的开发与应用。
Cell. 2014 Jun 5;157(6):1262-1278. doi: 10.1016/j.cell.2014.05.010.
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Generation of engraftable hematopoietic stem cells from induced pluripotent stem cells by way of teratoma formation.通过畸胎瘤形成从诱导多能干细胞生成可移植造血干细胞。
Mol Ther. 2013 Jul;21(7):1424-31. doi: 10.1038/mt.2013.71. Epub 2013 May 14.
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In vivo generation of transplantable human hematopoietic cells from induced pluripotent stem cells.从诱导多能干细胞生成可移植的人类造血细胞。
Blood. 2013 Feb 21;121(8):1255-64. doi: 10.1182/blood-2012-06-434407. Epub 2012 Dec 4.