Feasibility of gene therapy for Friedreich Ataxia-associated cardiomyopathy in non-human primates: Evaluation of delivery route, biodistribution and expression following AAVRh.10.FXN administration

被引：0

|

作者：

Belbellaa, B. ^{[1
]}

Bonnamain, V. ^{[1
]}

Kaminsky, S. M. ^{[2
]}

Sondhi, D. ^{[2
]}

Rosenberg, J. ^{[2
]}

Kozarsky, K. ^{[3
]}

Puccio, H. ^{[4
,5
,6
,7
]}

Crystal, R. G. ^{[2
]}

Gasmi, M. ^{[1
]}

机构：

[1] Adverum Biotechnol Inc, Menlo Pk, CA USA

[2] Weill Cornell Med, Dept Med Genet, New York, NY USA

[3] Vector BioPartners, Bala Cynwyd, PA USA

[4] Inst Genet & Biol Mol & Cellulaire, Dept Translat Med & Neurogenet, Illkirch Graffenstaden, France

[5] INSERM, U1258, Illkirch Graffenstaden, France

[6] CNRS, UMR7104, Illkirch Graffenstaden, France

[7] Univ Strasbourg, Strasbourg, 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：

P480

引用

收藏

页码：A158 / A159

页数：2

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