AAV-mediated gene therapy shows efficacy on the biochemical and neurological phenotype of the nucleoside-enhanced mouse model of MNGIE

被引：0
|
作者：
Vila-Julia, F. [1 ,2 ]
Cabrera-Perez, R. [1 ,2 ]
Lope-Piedrafita, S. [3 ,4 ]
Camara, Y. [1 ,2 ]
Hirano, M. [5 ]
Mingozzi, F. [6 ,7 ,8 ]
Torres-Torronteras, J. [1 ,2 ]
Marti, R. [1 ,2 ]
机构：
[1] Univ Autonoma Barcelona, Vall dHebron Res Inst, Res Grp Neuromuscular & Mitochondrial Dis, Barcelona, Catalonia, Spain
[2] Inst Salud Carlos III, Biomed Network Res Ctr Rare Dis, Madrid, Spain
[3] Univ Autonoma Barcelona, Nucl Magnet Resonance Serv, Cerdanyola Del Valles, Catalonia, Spain
[4] Univ Autonoma Barcelona, Biomed Network Res Ctr Bioengn Biomat & Nanomed, Cerdanyola Del Valles, Catalonia, Spain
[5] Columbia Univ, Dept Neurol, Med Ctr, H Houston Merritt Neuromuscular Res Ctr, New York, NY USA
[6] Genethon, Evry, France
[7] INSERM, U951, Evry, France
[8] Univ Pierre & Marie Curie, Paris, France
来源：
HUMAN GENE THERAPY | 2019年 / 30卷 / 11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：
P442
引用
收藏
页码：A147 / A148
页数：2
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