Instances of duschene muscular dystrophy

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Remember that many instances of Duschene's Muscular Dystrophy are caused by a stop mutation in an exon of the gene dystrophin, and that the disease symptoms can be alleviated by skipping that exon (refer to the pre-mRNA processing lecture if needed).

With this general idea in mind, outline a CRISPR-based approach for curing muscular dystrophy that targets the dystrophin gene. Focus on the genetic modification, not on peripheral issues like delivering the CRISPR system to muscles (this is actually doable using genetically modified adenovirus!).

Reference no: EM132695697

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