Thursday, November 24, 2016

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.

                                                                                  Image result for duchenne muscular dystrophy

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.




1 comment:

  1. Muscular dystrophy is a very serious issue. Because the people who have it lose muscular ability, they are often confined to a wheelchair as their muscles continue to deteriorate. I knew somebody who died recently from muscular dystrophy, at the young age of 18. The development of the microgene talked about in this article can help slow the progression of the disease so that those affected with muscular dystrophy can live longer, healthier lives, and possibly even move around freely without the use of a wheelchair if treated early on.