A Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular Dystrophy

被引：0

作者：

Mendell, Jerry R. ^{[1
,2
]}

Shieh, Perry B. ^{[3
]}

Sahenk, Zarife ^{[1
]}

Lehman, Kelly ^{[1
]}

Lowes, Linda P. ^{[1
]}

Reash, Natalie F. ^{[1
]}

Iammarino, Megan ^{[1
]}

Alfano, Lindsay N. ^{[1
]}

Woods, Jeremy D. ^{[3
]}

Skura, Christy L. ^{[3
]}

Mao, Howard C. ^{[3
]}

Staudt, Loretta A. ^{[3
]}

Potter, Rachael A. ^{[1
,4
]}

Griffin, Danielle ^{[1
,4
]}

Lewis, Sarah ^{[1
,4
]}

Hu, Larry ^{[4
]}

Upadhyay, Sameer ^{[4
]}

Singh, Teji ^{[4
]}

Rodino-Klapac, Louise R. ^{[4
]}

机构：

[1] Nationwide Childrens Hosp, Res Inst, Ctr Gene Therapy, Columbus, OH USA

[2] Ohio State Univ, Dept Pediat & Neurol, Columbus, OH 43210 USA

[3] Ronald Reagan UCLA Med Ctr, Los Angeles, CA USA

[4] Sarepta Therapeut Inc, Cambridge, MA USA

来源：

MOLECULAR THERAPY | 2021年 / 29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：

231

引用

页码：120 / 121

页数：2

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