Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy

Version 1 : Received: 3 March 2020 / Approved: 4 March 2020 / Online: 4 March 2020 (04:58:48 CET)

A peer-reviewed article of this Preprint also exists.

Lim, K.R.Q.; Nguyen, Q.; Dzierlega, K.; Huang, Y.; Yokota, T. CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy. Genes 2020, 11, 342. Lim, K.R.Q.; Nguyen, Q.; Dzierlega, K.; Huang, Y.; Yokota, T. CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy. Genes 2020, 11, 342.

Journal reference: Genes 2020, 11, 342
DOI: 10.3390/genes11030342

Abstract

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 quite well, each comes with their own limitations. To help overcome this, clustered regularly interspaced short palindromic repeats (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.

Subject Areas

Duchenne muscular dystrophy; CRISPR; animal models; in vivo testing; dystrophin; mutant generation

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