Treatment-Emergent Adverse Events in Gene Therapy Trials for Inherited Retinal Diseases: A Narrative Review

被引：19

作者：

Nuzbrokh, Yan ^{[1
,2
,3
]}

Kassotis, Alexis S. ^{[1
]}

Ragi, Sara D. ^{[1
,2
]}

Jauregui, Ruben ^{[1
,2
]}

Tsang, Stephen H. ^{[1
,2
,4
]}

机构：

[1] Columbia Univ, Irving Med Ctr, Edward S Harkness Eye Inst, Dept Ophthalmol, New York, NY 10027 USA

[2] Jonas Childrens Vis Care, New York, NY USA

[3] SUNY Stony Brook, Renaissance Sch Med, Stony Brook, NY 11794 USA

[4] Columbia Univ, Irving Med Ctr, Dept Pathol & Cell Biol, New York, NY USA

来源：

OPHTHALMOLOGY AND THERAPY | 2020年 / 9卷 / 04期

基金：

美国国家卫生研究院;

关键词：

Adverse events; Inherited retinal diseases; Retinal gene therapy; PARS-PLANA VITRECTOMY; X-LINKED RETINOSCHISIS; INTRAOCULAR-PRESSURE CHANGES; EYELID RETRACTION TECHNIQUE; LONG-TERM EFFICACY; INTRAVITREAL INJECTION; 25-GAUGE VITRECTOMY; POSTOPERATIVE HYPOTONY; SUTURELESS VITRECTOMY; ADENOASSOCIATED VIRUS;

D O I：

10.1007/s40123-020-00287-1

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

Patient safety is a primary priority in the conduction of retinal gene therapy trials. An understanding of risk factors and mitigation strategies for post-procedure complications is crucial for the optimization of gene therapy clinical trial protocols. In this review, we synthesize the literature on ocular delivery methods, vector platforms, and treatment-emergent adverse effects in recent gene therapy clinical trials for inherited retinal diseases.

引用

页码：709 / 724

页数：16

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