CRISPR/Cas correction of muscular dystrophies

被引：16

作者：

Zhang, Yu ^{[1
,2
]}

Nishiyama, Takahiko ^{[1
,2
]}

Olson, Eric N. ^{[1
,2
]}

Bassel-Duby, Rhonda ^{[1
,2
]}

机构：

[1] Univ Texas Southwestern Med Ctr Dallas, Dept Mol Biol, Dallas, TX 75390 USA

[2] Univ Texas Southwestern Med Ctr Dallas, Senator Paul D Wellstone Muscular Dystrophy Speci, Dallas, TX 75390 USA

来源：

EXPERIMENTAL CELL RESEARCH | 2021年 / 408卷 / 01期

关键词：

Genome editing; sgRNA; Duchenne muscular dystrophy; Facioscapulohumeral muscular dystrophy; Limb-girdle muscular dystrophy; Skeletal muscle; MOUSE MODEL; CRISPR-CAS9; NUCLEASES; MYOTONIC-DYSTROPHY; STEM-CELLS; MUSCLE; GENE; MUTATIONS; DNA; DEFICIENCY; EXPRESSION;

D O I：

10.1016/j.yexcr.2021.112844

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Muscular dystrophies are a heterogeneous group of monogenic neuromuscular disorders which lead to progressive muscle loss and degeneration of the musculoskeletal system. The genetic causes of muscular dystrophies are well characterized, but no effective treatments have been developed so far. The discovery and application of the CRISPR/Cas system for genome editing offers a new path for disease treatment with the potential to permanently correct genetic mutations. The post-mitotic and multinucleated features of skeletal muscle provide an ideal target for CRISPR/Cas therapeutic genome editing because correction of a subpopulation of nuclei can provide benefit to the whole myofiber. In this review, we provide an overview of the CRISPR/Cas system and its derivatives in genome editing, proposing potential CRISPR/Cas-based therapies to correct diverse muscular dystrophies, and we discuss challenges for translating CRISPR/Cas genome editing to a viable therapy for permanent correction of muscular dystrophies.

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页数：9

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