Host research mentor: Michelle Calos, Ph.D.
Stanford University
The Calos Lab is focused on developing a combined gene therapy and stem cell therapy technique in order to treat Duchenne Muscular Dystrophy (DMD). DMD is a common and fatal genetic disease caused by a mutation in the dystrophin gene. We are seeking to reprogram fibroblasts from afflicted patients and correct the disease by addition of functioning dystrophin. Using a bacterial integrase known as PhiC31, we are inserting a removable reprogramming cassette and the therapeutic gene stably into the genome. Once reprogramming to to induced pluripotent stem cells is achieved, gene corrected cells can be pushed down a differentiation path to muscle precursor cells. Utilizing this method, we aim to create corrected muscle precursor cells with a single copy of the gene of interest integrated into a safe location within the genome. These muscle precursor cells can then be engrafted into the afflicted patient and alleviate the symptoms of the disease. This study encompasses mouse, dog, and human DMD models.