Danon Disease Phase 1 RP-A501 Results: The First Single-Dose Intravenous (IV) Gene Therapy with Recombinant Adeno-Associated Virus (AAV9:LAMP2B) for a Monogenic Cardiomyopathy

被引：0
|
作者：
Greenberg, Barry [1 ]
Rossano, Joseph [2 ]
Taylor, Matthew [3 ]
Adler, Eric D. [1 ]
Lin, Kimberly Y. [2 ]
Eshraghian, Emily [1 ]
Attias, Jennifer [1 ]
Hong, Kimberly N. [1 ]
Urey, Marcus Anthony [1 ]
Shah, Monica [4 ]
Ricks, David [4 ]
Yarabe, Paul [4 ]
Schwartz, Jonathan [4 ]
机构：
[1] Univ Calif La Jolla, San Diego Med Ctr, La Jolla, CA USA
[2] Childrens Hosp Philadelphia, Philadelphia, PA USA
[3] Univ Colorado Anschutz Med Campus, Aurora, CO USA
[4] Rocket Pharmaceut Inc, Cranbury, NJ USA
来源：
MOLECULAR THERAPY | 2023年 / 31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：
9
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收藏
页码：6 / 6
页数：1
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