Genes, Vol. 11, Pages 342: CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy

Genes, Vol. 11, Pages 342: CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy Genes doi: 10.3390/genes11030342 Authors: Lim Nguyen Dzierlega Huang Yokota 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.
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Review Source Type: research