Developing AAV-Mediated Clinically Translatable Gene Therapy for Maple Syrup Urine Disease (MSUD) Caused by BCKDHA Mutations in a Bovine Model

被引：0
|
作者：
Wang, Jiaming [1 ,2 ]
Wang, Dan [1 ,2 ]
Beever, Jonathan [3 ]
Puffenberger, Erik [4 ]
GrayEdwards, Heather [1 ]
Strauss, Kevin A. [4 ]
Gao, Guangping [1 ,2 ,5 ]
机构：
[1] Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA USA
[2] Univ Massachusetts, Sch Med, Dept Microbiol & Physiol Syst, Worcester, MA USA
[3] Univ Illinois, Dept Anim Sci, 328 Mumford Hall, Urbana, IL 61801 USA
[4] Clin Special Children, Strasburg, PA USA
[5] Univ Massachusetts, Sch Med, Li Weibo Inst Rare Dis Res, Worcester, MA USA
来源：
MOLECULAR THERAPY | 2019年 / 27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：
505
引用
收藏
页码：238 / 239
页数：2
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