Gene therapy for neurological disorders: progress and prospects

被引：0
作者：
Benjamin E. Deverman
Bernard M. Ravina
Krystof S. Bankiewicz
Steven M. Paul
Dinah W. Y. Sah
机构：
[1] California Institute of Technology,Division of Biology and Biological Engineering
[2] Voyager Therapeutics,Department of Neurological Surgery
[3] University of California–San Francisco,undefined
[4] Present address: Stanley Center for Psychiatric Research at Broad Institute,undefined
[5] Cambridge,undefined
[6] MA,undefined
[7] USA.,undefined
来源：
Nature Reviews Drug Discovery | 2018年 / 17卷
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摘要：
The potential of adeno-associated viral (AAV)-mediated gene therapy for neurological disorders is rapidly emerging. Evidence of clinical efficacy and safety, as well as durable transgene expression, has now been reported in several central nervous system disorders. Here, Sah and colleagues discuss key considerations in the design and development of therapeutic AAV vectors, highlighting promising therapeutic targets and recent clinical trials.
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页码：641 / 659
页数：18
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