Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases

被引：86

作者：

Wu, Shao-Shuai ^{[1
]}

Li, Qing-Cui ^{[1
]}

Yin, Chang-Qing ^{[1
]}

Xue, Wen ^{[2
,3
,4
]}

Song, Chun-Qing ^{[1
]}

机构：

[1] Westlake Univ, Sch Life Sci, Key Lab Growth Regulat & Transformat Res Zhejiang, 18 Shilongshan Rd, Hangzhou 310024, Zhejiang, Peoples R China

[2] Univ Massachusetts, Med Sch, RNA Therapeut Inst, Worcester, MA 01605 USA

[3] Univ Massachusetts, Med Sch, Program Mol Med, Worcester, MA 01605 USA

[4] Univ Massachusetts, Med Sch, Dept Mol Cell & Canc Biol, Worcester, MA 01605 USA

来源：

THERANOSTICS | 2020年 / 10卷 / 10期

基金：

美国国家卫生研究院;

关键词：

CRISPR/Cas; Gene editing; Gene therapy; Human disease; Genetic disease; IN-VIVO; MOUSE MODEL; ADAPTIVE IMMUNITY; CYSTIC-FIBROSIS; GENOME; CRISPR-CAS9; GENERATION; MUSCLE; CELLS; CAS;

D O I：

10.7150/thno.43360

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

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.

引用

页码：4374 / 4382

页数：9

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