Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy is a particularly virulent form of muscular dystrophy that quickly weakens muscles throughout the entire body due to the resulting lack of a functional dystrophin protein that is integral for muscular function. It has a very early onset and spreads quickly, often debilitating the recipients and forcing them to be wheelchair bound well before adulthood. Dystrophin is essential for muscle to maintain its integrity and lacking the particular protein will sadly force an individual to waste away and be plagued with health problems for the rest of their life (which will also be shortened to around 30 years). The onset can begin very early, even in infancy as the child's muscles will fail to properly develop resulting in difficulty with mobility. It generally takes a child with DMD significantly longer to learn to sit, crawl, or walk. Later in adolescence, they will develop a gait in their step and will have difficulty moving through any sort of rough terrain. The disease continues to worsen throughout the recipient's life as muscle tissue swells and will progressively be replaced with adipose and scar tissue. This primarily effects the legs first, but will worsen in other parts of the body as well, even 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, aka “DMD” is an inherited disorder that results from a mutation in the gene that regulates production of the dystrophin protein located within the X-chromosome. It stands out from most other forms of dystrophy due to its aggressive degeneration of muscle tissue and very early onset. Thankfully this disorder is a recessive trait and therefore ...

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...have been met with some success in trials with mice. While I'm not certain how this could be effectively implemented throughout the entirety of an individual's body I do take comfort in the fact that there are many far more knowledgeable in this field than myself who may very well find a solution to that issue. At the very least perfecting methods of gene transfer via viral transmission or some other means could potentially be used on fertilized egg cells in the early stages of life and in doing so, effectively eliminate the problem at 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 faulty genes from tainted chromosomes can be safely prevented from passing onto the next generation without denying individuals the right to reproduce. I hope that day comes sooner than later.