腺相关病毒载体在视网膜中用于CRISPR介导的体内基因组编辑
Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina.
作者信息
Yu Wenhan, Wu Zhijian
机构信息
Ocular Gene Therapy Core, National Eye Institute, NIH, Bethesda, MD, USA.
出版信息
Methods Mol Biol. 2019;1950:123-139. doi: 10.1007/978-1-4939-9139-6_7.
Abstract
Degenerative retinal diseases such as retinitis pigmentosa (RP) and Leber's congenital amaurosis (LCA) may lead to blindness without effective treatment. With the rapid advancement of the CRISPR/Cas9 genome editing technology, in vivo application of CRISPR/Cas9 holds immense potential for treatment of these diseases. Adeno-associated virus (AAV) vectors are an ideal gene transfer tool for delivery of CRISPR components to the retina. Here, we describe a protocol for utilizing an AAV-based CRISPR/Cas9 system for in vivo genome editing in the retina.
摘要
诸如色素性视网膜炎（RP）和莱伯先天性黑矇（LCA）等退行性视网膜疾病若没有有效的治疗方法，可能会导致失明。随着CRISPR/Cas9基因组编辑技术的迅速发展，CRISPR/Cas9在体内的应用为治疗这些疾病具有巨大潜力。腺相关病毒（AAV）载体是将CRISPR组件递送至视网膜的理想基因传递工具。在此，我们描述了一种利用基于AAV的CRISPR/Cas9系统在视网膜中进行体内基因组编辑的方案。
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