Gene-editing therapy for neurological disease

被引：22
|
作者：
McMahon, Moira A. [1 ,2 ]
Cleveland, Don W. [1 ,2 ]
机构：
[1] Univ Calif San Diego, Ludwig Inst, 9500 Gilman Dr, La Jolla, CA 92093 USA
[2] Univ Calif San Diego, Dept Cellular & Mol Med, 9500 Gilman Dr, La Jolla, CA 92093 USA
来源：
NATURE REVIEWS NEUROLOGY | 2017年 / 13卷 / 01期
关键词：
D O I：
10.1038/nrneurol.2016.190
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
Guide RNA-mediated CRISPR-Cas nucleases are a powerful technology for the engineering of mammalian genomes. CRISPR-Cas9-dependent editing of mutated genes that cause Huntington disease and fragile X syndrome was recently achieved in cell-based models, heralding the first step towards developing this technology into viable therapeutics for neurological diseases.
引用
收藏
页码：7 / 9
页数：3
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