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 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.
Highlights
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disorder characterized by progressive muscle degeneration and weakness [1]
All these improvements are made possible by the small number of reagents required, the ease of reagent delivery, the high efficiency associated with clustered regularly interspaced short palindromic repeat (CRISPR), and the bypassing of complex steps that characterized earlier methods of transgenic animal creation, such as the need for using embryonic stem cells and extensive breeding strategies [58,61,62]
We have extensively reviewed the utility of CRISPR in developing genome-editing therapies for DMD [33]
Summary
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disorder characterized by progressive muscle degeneration and weakness [1]. The mdx mouse shows much milder phenotypes, with no severe cardiac involvement; lacks a pronounced effect of the disease on survival; has regenerative and compensatory mechanisms in response to dystrophin loss; and represents a single mutation out of the possible thousands found in patients [22,23,24,25,26]. When used with nucleases such as CRISPR-associated protein 9 (Cas9), CRISPR allows for the precise editing of virtually any target gene [31,32] With such capabilities, CRISPR has revolutionized the DMD field, as an alternative therapeutic strategy for the disorder and in providing new in vitro and in vivo DMD models [33]. We conclude with some challenges and future prospects on the use of these in vivo models in the field
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