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Open AccessReview

CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy

Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada
The Friends of Garrett Cumming Research & Muscular Dystrophy Canada, HM Toupin Neurological Science Research Chair, Edmonton, AB T6G 2H7, Canada
Author to whom correspondence should be addressed.
Genes 2020, 11(3), 342;
Received: 2 March 2020 / Revised: 21 March 2020 / Accepted: 23 March 2020 / Published: 24 March 2020
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disorder most commonly caused by mutations disrupting the reading frame of the dystrophin (DMD) gene. DMD codes for dystrophin, which is critical for maintaining the integrity of muscle cell membranes. Without dystrophin, muscle cells receive heightened mechanical stress, becoming more susceptible to damage. An active body of research continues to explore therapeutic treatments for DMD as well as to further our understanding of the disease. These efforts rely on having reliable animal models that accurately recapitulate disease presentation in humans. While current animal models of DMD have served this purpose well to some extent, each has its own limitations. To help overcome this, clustered regularly interspaced short palindromic repeat (CRISPR)-based technology has been extremely useful in creating novel animal models for DMD. This review focuses on animal models developed for DMD that have been created using CRISPR, their advantages and disadvantages as well as their applications in the DMD field. View Full-Text
Keywords: Duchenne muscular dystrophy; CRISPR; animal models; in vivo testing; dystrophin; mutant generation Duchenne muscular dystrophy; CRISPR; animal models; in vivo testing; dystrophin; mutant generation
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Lim, K.R.Q.; Nguyen, Q.; Dzierlega, K.; Huang, Y.; Yokota, T. CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy. Genes 2020, 11, 342.

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