Gene therapy for neurological disorders: progress and prospects

被引：218

作者：

Deverman, Benjamin E. ^{[1
,4
]}

Ravina, Bernard M. ^{[2
]}

Bankiewicz, Krystof S. ^{[3
]}

Paul, Steven M. ^{[2
]}

Sah, Dinah W. Y. ^{[2
]}

机构：

[1] CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

[2] Voyager Therapeut, Cambridge, MA USA

[3] Univ Calif San Francisco, Dept Neurol Surg, San Francisco, CA 94143 USA

[4] Broad Inst, Stanley Ctr Psychiat Res, Cambridge, MA USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2018年 / 17卷 / 09期

关键词：

ADENOASSOCIATED VIRUS TYPE-2; CENTRAL-NERVOUS-SYSTEM; AMYOTROPHIC-LATERAL-SCLEROSIS; ADVANCED PARKINSONS-DISEASE; SEVERE MENTAL-RETARDATION; SPINAL-CORD TRANSDUCTION; BLOOD-BRAIN-BARRIER; RATE-LIMITING STEP; MOUSE MODEL; VIRAL VECTOR;

D O I：

10.1038/nrd.2018.110

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic proteins, microRNAs, antibodies or gene-editing machinery have been successfully delivered to the central nervous system with natural or engineered viral capsids via various routes of administration. Importantly, initial clinical studies have demonstrated encouraging safety and efficacy in diseases such as Parkinson disease and spinal muscular atrophy, as well as durability of transgene expression. Here, we discuss key considerations and challenges in the future design and development of therapeutic AAV vectors, highlighting the most promising targets and recent clinical advances.

引用

页码：641 / 659

页数：19

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