Affiliations: Biology, Psychological and Brain Sciences, Neuroscience
Award: Woodrow Wilson
Application of CRISPR-on technology to up-regulate utrophin expression as therapy for Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle weakness and muscle wasting. DMD is caused by mutations that lead to deficiency in dystrophin, a protein that is critical for maintaining muscle health. One recently considered method of gene therapy focuses on the replacement of dystrophin with a similar protein, utrophin. I investigated the application of the microbial-based genome modifying system, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR), to efficiently target and activate utrophin expression in a mouse cell line model for DMD.
Mentors: Dr. Ronald Cohn, Department of Pediatrics, SOM, and Dr. Amanda Bergner, Department of Neurology, SOM