CRISPR technologies for the treatment of Duchenne muscular dystrophy

被引：37

作者：

Choi, Eunyoung ^{[1
]}

Koo, Taeyoung ^{[1
,2
,3
]}

机构：

[1] Kyung Hee Univ, Dept Life & Nanopharmaceut Sci, Seoul, South Korea

[2] Kyung Hee Univ, Grad Sch, Dept Biomed & Pharmaceut Sci, Seoul, South Korea

[3] Kyung Hee Univ, Coll Pharm, Dept Pharmaceut Sci, Seoul 02447, South Korea

来源：

MOLECULAR THERAPY | 2021年 / 29卷 / 11期

基金：

新加坡国家研究基金会;

关键词：

UTROPHIN UP-REGULATION; MOUSE MODEL; NONHUMAN-PRIMATES; GENE ACTIVATION; SEVERE TOXICITY; GENOMIC DNA; STEM-CELLS; MUSCLE; BASE; MICE;

D O I：

10.1016/j.ymthe.2021.04.002

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated genome editing technologies have progressed remarkably in recent years, opening up the potential of precise genome editing as a therapeutic approach to treat various diseases. The CRISPR-CRISPR-associated (Cas) system is an attractive platform for the treatment of Duchenne muscular dystrophy (DMD), which is a neuromuscular disease caused by mutations in the DMD gene. CRISPR-Cas can be used to permanently repair the mutated DMD gene, leading to the expression of the encoded protein, dystrophin, in systems ranging from cells derived from DMD patients to animal models of DMD. However, the development of more efficient therapeutic approaches and delivery methods remains a great challenge for DMD. Here, we review various therapeutic strategies that use CRISPR-Cas to correct or bypass DMD mutations and discuss their therapeutic potential, as well as obstacles that lie ahead.

引用

页码：3179 / 3191

页数：13

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