Start Date: October 1, 2019
George Kargas
Duchenne’s muscular dystrophy is an incurable disease caused by a dystrophin gene mutation. The mutation results in dystrophin deficiency and gradual muscle damage. Gene therapies for the disease have recently been developed, which have had positive results in laboratory and clinical trials. One approach is to enrich the muscle cells with genes that can produce functional dystrophin. Genetic material is transferred to cells by adenovirus-associated viruses. In the context of the proposed work, a computational method for guide RNA (gRNA) selection will be developed with the ultimate goal of altering the protein sequence that expresses the dystrophin gene. Guide RNAs are the RNAs that guide the insertion or deletion of uridine residues in mitochondrial mRNAs in kinetoplastid reactions in a process known as RNA processing. The terms “guide RNA” and “gRNA” are also used in prokaryotic DNA processing which includes the CRISPR and Cas9 gene processing method. This method will be based on our knowledge of the structure and function of dystrophin.