Design of a Phase 1/2 Study to Evaluate the Safety and Efficacy of an AAV9-Based Gene Therapy in Infants with Type 2 Gaucher Disease

被引：0
|
作者：
Youssef, Eriene [1 ]
Tamburri, Paul [1 ]
Beckerman, Yael [1 ]
Lowrey, Mark [1 ]
Mahoney, Erin [1 ]
McNamara, Ilan [1 ]
Sevigny, Jeffrey [1 ]
机构：
[1] Prevail Therapeut, New York, NY USA
来源：
MOLECULAR THERAPY | 2020年 / 28卷 / 04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：
623
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收藏
页码：276 / 277
页数：2
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