AAV8-Mediated Liver-Directed Gene Therapy as a Potential Therapeutic Option in Adults with Glycogen Storage Disease Type Ia (GSDIa): Results from a Phase 1/2 Clinical Trial

被引：0
|
作者：
Weinstein, David A. [1 ]
Ahmad, Ayesha [2 ]
Rodriguez-Buritica, David F. [3 ]
Mitchell, John [4 ]
Derks, Terry G. [5 ]
Mou, Jiani [6 ]
Poma, Allen [6 ]
Crombez, Eric [6 ]
机构：
[1] Univ Connecticut, Farmington, CT USA
[2] Univ Michigan, Ann Arbor, MI 48109 USA
[3] Univ Texas McGovern Med Sch, Houston, TX USA
[4] Montreal Childrens Hosp, Montreal, PQ, Canada
[5] Univ Groningen, Groningen, Netherlands
[6] Ultragenyx Gene Therapy, Cambridge, MA USA
来源：
MOLECULAR THERAPY | 2020年 / 28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：
1306
引用
收藏
页码：558 / 558
页数：1
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