Endogenous Human SMN1 Promoter-driven Gene Replacement Improves the Efficacy and Safety of AAV9-mediated Gene Therapy for SMA in mice

被引：0
|
作者：
Xie, J. [1 ]
Xie, Q. [1 ]
Ma, H. [1 ]
Chen, X. [1 ]
Zhu, Y. [2 ]
Ma, Y. [2 ]
Jalinous, L. [2 ]
Su, Q. [1 ]
Tai, P. [1 ]
Gao, G. [1 ]
机构：
[1] Umass Chan Med Sch, Gene Therapy Ctr, Worcester, MA USA
[2] CANbridge Pharmaceut, Cambridge, MA USA
来源：
NEUROMUSCULAR DISORDERS | 2022年 / 32卷
关键词：
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暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
LSVP.05
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页数：1
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