In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

出版年份 2019 全文链接

标题

作者

关键词

sickle cell disease, gene editing, rAAV6, ssODN, homology-directed repair, Crispr/Cas9, hemoglobin disorders, NHEJ versus HDR, in vivo, engraftment, NBSGW41 mice, CD34, hematopoietic stem cells, stem cell cures

出版物

Molecular Therapy-Nucleic Acids

Volume 17, Issue -, Pages 277-288

出版商

Elsevier BV

发表日期

2019-06-08

DOI

10.1016/j.omtn.2019.05.025

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