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Theoretical Applicability Of Crispr-Mediated Exon Skipping In Duchenne Muscular Dystrophy, Andrew Greek

Biomedical Engineering Undergraduate Honors Theses

Duchenne muscular dystrophy (DMD) affects approximately 1 in 5,000 males. DMD results from genetic mutations within the gene that interrupts the open reading frame and prematurely truncates the protein. From a non-functional dystrophin protein, DMD results in serious muscle weakening and muscle wasting ultimately leading to death around the age of 26. In some cases of muscular dystrophy, a mutation can occur in areas of the gene that are less essential. In Becker muscular dystrophy (BMD), these mutations cause less significant consequences in phenotype as the dystrophin protein is still semi-functional. With gene editing techniques, it may be possible to …