AAV.U7 Induced Exon Skipping for a Mutational Hotspot (∼6%) of the DMD Gene Results in Efficient Exon Skipping, Protein Restoration and Force Improvement

被引：0
|
作者：
Rajakumar, Dhanarajan [1 ]
Lesman, Daniel [1 ]
Li, Ding [1 ]
Gaffney, Caleb [1 ]
Rafferty, Rachel [1 ]
Huang, Nianyuan [1 ]
Rodriguez, Yacidzohara [1 ]
Young, Courtney [2 ,3 ]
Spencer, Melissa [2 ,3 ]
Flanigan, Kevin [1 ,4 ]
Wein, Nicolas [1 ,4 ]
机构：
[1] Nationwide Childrens Hosp, Ctr Gene Therapy, Abigail Wexner Res Inst, Columbus, OH USA
[2] Univ Calif Los Angeles, Inst Mol Biol, Los Angeles, CA 90024 USA
[3] Univ Calif Los Angeles, Dept Neurol, Los Angeles, CA 90024 USA
[4] Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA
来源：
MOLECULAR THERAPY | 2020年 / 28卷 / 04期
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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：
1069
引用
收藏
页码：463 / 463
页数：1
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