Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques

被引：6

作者：

Cho, Galaxy Y. ^{[1
,2
,3
,4
]}

Bolo, Kyle ^{[5
]}

Park, Karen Sophia ^{[1
,2
,3
]}

Sengillo, Jesse D. ^{[6
]}

Tsang, Stephen H. ^{[1
,2
,3
,5
,7
,8
]}

机构：

[1] Jonas Childrens Vis Care, New York, NY 10032 USA

[2] Bernard & Shirlee Brown Glaucoma Lab, New York, NY 10032 USA

[3] Columbia Univ, Dept Ophthalmol, New York, NY 10027 USA

[4] Quinnipiac Univ, Frank H Netter MD Sch Med, North Haven, CT USA

[5] Columbia Univ, Vagelos Coll Phys & Surg, New York, NY 10032 USA

[6] Reading Hosp, Dept Internal Med, W Reading, PA USA

[7] Inst Human Nutr, Dept Pathol & Cell Biol, Stem Cell Initiat CSCI, New York, NY 10032 USA

[8] Columbia Univ, Med Ctr, Harkness Eye Inst, 635 West 165th St,Box 212, New York, NY 10032 USA

来源：

MOLECULAR DIAGNOSIS & THERAPY | 2019年 / 23卷 / 01期

基金：

美国国家卫生研究院;

关键词：

LEBER CONGENITAL AMAUROSIS; HEREDITARY OPTIC NEUROPATHY; ADENOASSOCIATED VIRUS; MACULAR DEGENERATION; SUBRETINAL INJECTION; RETINITIS-PIGMENTOSA; RPE65; MUTATIONS; IMMUNE-RESPONSE; OPEN-LABEL; FOLLOW-UP;

D O I：

10.1007/s40291-018-0377-1

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Inherited retinal dystrophies cause progressive vision loss and are major contributors to blindness worldwide. Advances in gene therapy have brought molecular approaches into the realm of clinical trials for these incurable illnesses. Select phase I, II and III trials are complete and provide some promise in terms of functional outcomes and safety, although questions do remain over the durability of their effects and the prevalence of inflammatory reactions. This article reviews gene therapy as it can be applied to inherited retinal dystrophies, provides an update of results from recent clinical trials, and discusses the future prospects of gene therapy and genome surgery.

引用

页码：113 / 120

页数：8

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