AAV8-hLCA5 Subretinal Delivery to the Macula in LCA5-associated Leber Congenital Amaurosis (LCA5-LCA): Preliminary Safety and Efficacy Results of an Ongoing Phase I/II Gene Therapy Trial

被引：0

|

作者：

Aleman, Tomas S. ^{[1
]}

Cideciyan, Artur V. ^{[1
]}

Weber, Mariejel L. ^{[1
]}

Santos, Arlene ^{[1
]}

Uyhazi, Katherine E. ^{[1
]}

Parchinski, Kelsey ^{[1
]}

Chung, William ^{[1
]}

Margaritis, Paris ^{[1
]}

Sun, Junwei ^{[1
]}

Scoles, Drew ^{[1
]}

Garafalo, Alexandra V. ^{[1
]}

Jayagopal, Ashwath ^{[2
]}

Yerxa, Benjamin R. ^{[2
]}

Tuller, Sarah ^{[2
]}

Maguire, Albert ^{[1
]}

Bennett, Jean ^{[1
]}

机构：

[1] Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA USA

[2] Opus Genet, Durham, NC USA

来源：

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2024年 / 65卷 / 07期

基金：

美国国家卫生研究院;

关键词：

D O I：

暂无

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

3288

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收藏

页数：3

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