Promise in Tackling Muscular Dystrophy

Muscular Dystrophy, is “when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function”¹ has impacted around 250,000 people in the United States, and Duchenne Muscular Dystrophy (DMD) only affects boys. Recent clinical studies have been conducted on dogs to treat DMD during the early stages of development, and human trials are scheduled to begin in the next few years.

                                                                                  

Boys with DMD have a mutation in their genes that interrupt dystrophin protein production, and thus cause a phenotypic complication. The muscle cells degenerate, resulting in difficulty in mobility, such as walking and even breathing. Gene therapy has been unsuccessful in the past due to the large size of the gene, however past research has allowed the development of a microgene, in which a gene therapy vector can carry a micro-dystrophin to all muscles by way of a virus to offer protection of this disease.

This brings hope to boys who are showing early signs of Duchenne Muscular Dystrophy. With this microgene, there is promise for their muscle cells to be protected from this disease, as well as prevent further degeneration of mobility. This will be the beginning of these boys taking their life back, and truly living to the fullest.

Sources:

1. https://www.sciencedaily.com/releases/2015/10/151022141722.htm

2. http://munews.missouri.edu/news-releases/2015/1022-gene-therapy-treats-all-muscles-in-the-body-in-muscular-dystrophy-dogs-human-clinical-trials-are-next-step/