VY-NPC101: in vitro and in vivo validation of a novel AAV gene replacement therapy to treat Niemann-Pick disease Type C1

被引：0
|
作者：
Knoll, E. [1 ]
Ezell, B. [1 ]
Liu, E. [1 ]
Sayed-Zahid, A. [1 ]
Li, M. [1 ]
Patzke, H. [1 ]
Tocci, J. [1 ]
Brown, J. [1 ]
Bales, K. [1 ]
Yonutas, H. M. [1 ]
机构：
[1] Voyager Therapeut, Cambridge, MA USA
来源：
HUMAN GENE THERAPY | 2021年 / 32卷 / 19-20期
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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：
P071
引用
收藏
页码：A50 / A50
页数：1
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