Genome Edited Human Hematopoietic Stem Cells Correct Lysosomal Storage Disorders: Proof-of-Concept and Safety Studies for Mucopolysaccharidosis Type I and Gaucher Disease

被引：0
|
作者：
Gomez-Ospina, Natalia [1 ]
Scharenberg, Sam Glynne [1 ]
Mostrel, Nathalie [1 ]
Raj, Nitin [2 ]
Attardi, Laura [2 ]
Khan, Shaukat [3 ]
Tomatsu, Shunji [3 ]
Lee, Ciaran [4 ,5 ]
Bao, Gang [4 ,5 ]
Porteus, Matthew H. [1 ]
机构：
[1] Stanford, Pediat, Stanford, CA USA
[2] Stanford, Radiat Oncol, Stanford, CA USA
[3] Nemours Alfred I duPont Hosp Children, Skeletal Dysplasia, Orthoped & BioMed, Wilmington, DE USA
[4] Georgia Inst Technol, Biomed Engn, Atlanta, GA 30332 USA
[5] Emory Univ, Atlanta, GA 30322 USA
来源：
MOLECULAR THERAPY | 2019年 / 27卷 / 04期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
697
引用
收藏
页码：329 / 329
页数：1
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