Subretinal gene delivery using helper-dependent adenoviral vectors

被引：0
|
作者：
Linda Wu
Simon Lam
Huibi Cao
Rui Guan
Rongqi Duan
Derek van der Kooy
Rod Bremner
Robert S Molday
Jim Hu
机构：
[1] Hospital for Sick Children,Physiology and Experimental Medicine Program
[2] University of Toronto,Department of Laboratory Medicine and Pathobiology
[3] University of Toronto,Department of Paediatrics
[4] University of Toronto,Department of Molecular Genetics
[5] University Health Network,Toronto Western Research Institute
[6] University of Toronto,Department of Biochemistry and Molecular Biology
[7] 2350 Health Sciences Mall,undefined
[8] University of British Columbia,undefined
来源：
Cell & Bioscience | / 1卷
关键词：
Retinal Pigment Epithelial; Gene Therapy Trial; Retinal Pigment Epithelial Layer; Vector Dose; Subretinal Injection;
D O I：
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中图分类号：
学科分类号：
摘要：
This study describes the successful delivery of helper-dependent adenoviral vectors to the mouse retina with long term and robust levels of reporter expression in the retina without apparent adverse effects. Since these vectors have a large cloning capacity, they have great potential to extend the success of gene therapy achieved using the adeno-associated viral vector.
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