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：

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

页码：558 / 558

页数：1