Topic > Duchenne Muscular Dystrophy - 647

Duchenne Muscular Dystrophy is a particularly virulent form of muscular dystrophy that rapidly weakens muscles throughout the body due to the resulting lack of a functional protein dystrophin that is integral to muscle function . It has a very early onset and spreads rapidly, often debilitating recipients and causing them to be wheelchair-bound well before adulthood. Dystrophin is essential for the muscle to maintain its integrity and the lack of that particular protein will unfortunately force an individual to waste away and be plagued by health problems for the rest of their life (which will also be shortened to around 30 years). Onset can begin very early, even in childhood, as the child's muscles will fail to develop adequately, resulting in mobility difficulties. It generally takes a child with DMD much longer to learn to sit, crawl or walk. Later in adolescence, they will develop a walking gait and will have difficulty moving over any type of rough terrain. The disease continues to worsen throughout the recipient's life as the muscle tissue swells and will be progressively replaced by fat and scar tissue. This affects the legs first, but will also get worse in other parts of the body, including vital organs such as the lungs and heart. Death usually comes in the form of respiratory or heart failure around early adulthood. Duchenne muscular dystrophy, also known as "DMD", is an inherited disease that results from a mutation in the gene that regulates the production of the dystrophin protein located within the X chromosome. It differs from most other forms of dystrophy by the aggressive degeneration of muscle tissue and the very early onset. Luckily this disorder is a recessive trait and so... half the article... some successes have been found in experiments on mice. While I am not sure how this could be effectively implemented throughout an individual's body, I take comfort in the fact that there are many more experts than me in this field who may very well find a solution to this problem. At the very least, perfecting methods of gene transfer via viral transmission or other means could potentially be used on eggs fertilized early in life and, in so doing, effectively eliminate the problem at the time of conception. Perhaps in time we could see the genetic defect eliminated from the human gene pool altogether. It will be a bright day indeed when defective genes from contaminated chromosomes can be safely prevented from passing to the next generation without denying individuals the right to reproduce. I hope that day comes sooner or later.