基于 CRISPR/Cas 的基因治疗在人类遗传性疾病中的进展。
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases.
机构信息
Key Laboratory of Growth Regulation and Transformation Research of Zhejiang Province, School of Life Sciences, Westlake University, 18 Shilongshan Road, Hangzhou 310024, Zhejiang Province, China.
RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts.
出版信息
Theranostics. 2020 Mar 15;10(10):4374-4382. doi: 10.7150/thno.43360. eCollection 2020.
CRISPR/Cas genome editing is a simple, cost effective, and highly specific technique for introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non-homologous end joining, homology- directed repair, and single-base exchanges. Cas9/Cas12a nuclease, dCas9 transcriptional regulators, base editors, PRIME editors and RNA editing tools are widely used in basic research. Currently, a variety of CRISPR/Cas-based therapeutics are being investigated in clinical trials. Among many new findings that have advanced the field, we highlight a few recent advances that are relevant to CRISPR/Cas-based gene therapies for monogenic human genetic diseases.
CRISPR/Cas 基因组编辑是一种简单、经济高效且高度特异的遗传变异引入技术。在哺乳动物细胞中，CRISPR/Cas 可促进非同源末端连接、同源定向修复和单碱基交换。Cas9/Cas12a 核酸酶、dCas9 转录调控因子、碱基编辑器、Prime 编辑器和 RNA 编辑工具在基础研究中得到了广泛应用。目前，多种基于 CRISPR/Cas 的疗法正在临床试验中进行研究。在推动该领域发展的众多新发现中，我们重点介绍与基于 CRISPR/Cas 的单基因人类遗传疾病基因疗法相关的一些最新进展。
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