COHORT 1-4 RESULTS FROM A PHASE 1/2 CLINICAL TRIAL OF AN AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA (GSDIA)

被引：0
|
作者：
Rodriguez-Buritica, David F. [1 ]
Ahmad, Ayesha [2 ]
Pico, Maria-Luz Couce [3 ]
Derks, Terry G. [4 ]
Mitchell, John [5 ]
Riba-Wolman, Rebecca [6 ]
Weinstein, David A. [6 ]
Lee, Connie [7 ]
Valayannopoulos, Vassili [7 ]
Crombez, Eric [7 ]
机构：
[1] Univ Texas McGovern Med Sch, Houston, TX USA
[2] Univ Michigan, Ann Arbor, MI 48109 USA
[3] Hosp Clin Univ Santiago de Compostela, Santiago De Compostela, Spain
[4] Univ Groningen, Groningen, Netherlands
[5] Montreal Childrens Hosp, Montreal, PQ, Canada
[6] Univ Connecticut, Farmington, CT USA
[7] Ultragenyx Pharmaceut Inc, Novato, CA USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2022年 / 135卷 / 04期
关键词：
D O I：
暂无
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
76
引用
收藏
页码：296 / 296
页数：1
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