DMD, RIPK3, and MLKL gene editing by CRISPR Cas9 as myofiber protection against dystrophin deficiency and necroptosis in Duchenne muscular dystrophy: A literature review

Abstract


 BACKGROUND: Duchenne muscular dystrophy is a neuromuscular disease caused by a deficiency of dystrophin, which causes the skeletal and cardiac muscles to degenerate. Targeted deletion of DMD, RIPK3, and MLKL has been shown in several studies to prevent dystrophin deficiency and necroptosis, a critical hypothesis in the etiology of Duchenne muscular dystrophy. AIM: This research aimed to see if using CRISPR/Cas9 to target DMD, RIPK3, and MLKL is an effective therapeutic and if it has a long-term effect on Duchenne muscular dystrophy. METHODS: Abstracts and titles of articles were searched for specific keywords to summarize them using the method used in this study. The researcher will look over the entire article to see if it is valuable and relevant to the topic. RESULTS: CRISPR/Cas9-mediated genome editing in MDX mice can improve the primary genetic lesions that cause muscular dystrophy (DMD) and prevent disease development. Furthermore, Ripk3/Mlk1 double knockout completely blocked necroptosis susceptibility in necroptosis-sensitive cell lines, each to an indistinguishable degree. CONCLUSION: DMD, RIPK3, and MLKL gene editing by CRISPR/Cas9 is effective dystrophin insufficiency, sarcolemma fragility, poor intracellular signaling, myocyte death, inflammatory infiltration, muscle replacement, and necroptosis. However, more research is needed to determine its side effects and safety.