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
收藏
页码：709 / 724
页数：16
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