“In Duchenne muscular dystrophy, dystrophin is almost totally absent; the less dystrophin that is …show more content…
The main purpose for research to be conducted for this deadly disease is to slow the process of muscle degeneration in the skeletal muscles and to hopefully provide a solution of lack of dystrophin in many patients. “There are hundreds of mutations in the dystrophonin gene that can lead to the disease, but in 60 percent of people with Duchenne, their mutation will occur within a specific hot spot of the gene” (Vogt-James). The type of researching platform developed by UCLA scientists focuses on the one “hot spot” or the specific location where genetic mutation of the dystrophin gene occurs