Cause for Gene Therapy Hope

As I wrote in my essay about Ashley's Treatment, my younger brother was diagnosed with Duchenne's Muscular Dystrophy when he was in first grade. Since then, my parents have taken steps in order to maintain the strength of his muscles for as long as possible. My brother goes to physical therapy once a week and is also taking steroids. As a result of the treatment, my brother is still able to function normally, in that he is still able to walk and do things for himself, though not as easily as he once could. The only side effects of the treatment are quick mood swings, an increased appetite, and a reduction in his growth. None of these side effects are life threatening in the least and so the benefits of his treatment greatly outweigh the risks.

Recently, Janaiah Kota and some colleagues from the Center for Gene Therapy at Nationwide Children's Hospital in Columbus, Ohio engineered a virus which when injected into monkeys increased the monkeys' strength and also stopped the breakdown of muscle which is the main problem with my brother's disease. While this research is very promising and gives me hope that my brother one day may be able to lead a normal life, there are still risks with gene therapy which cannot be overlooked. The monkeys did not have a degenerative muscle disease and it is not known if this treatment will have any adverse effects in humans. If there is more research done on human subjects which still shows results without harmful risks, then I believe it could be considered an option for my brother. However, at the moment, my brother is young and enjoying life. Even though he cannot participate in certain activities, he finds things that make him happy and he is one of the funniest people I know. I would not want my brother to be another newspaper headline for the risks associated with gene therapy. Although I think gene therapy might ultimately help cure my brother of his disease, the unknown risks at the moment are too high. Hopefully this exciting development will progress quickly so that one day my brother will be able to enjoy a limitless life.

Relevant site: http://www.reuters.com/article/scienceNews/idUSTRE5AA3ZY20091111?feedType=RSS&feedName=scienceNews&rpc=69