Muscular Dystrophy Research Paper

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Good morning, today I am going to discuss the use of Gene Therapy on Muscular Dystrophy, and how Human manipulation of genes may be a dangerous endeavour because of the uncertainty of safe practices and the myriad of ethical issues that present both for present-day and future society. Gene Therapy is a controversial treatment for diseases as technology is not completely advanced to fully trust the results to come out successful on every disease and for every person. Although gene manipulation causes some uprise to the public on whether it is safe enough to use on humans, it has been proven in previous studies that gene therapy has been helpful to treat or prevent disease in people, and has majorly helped current research. In genetic modification, …show more content…
Chamberlain, 2002), which leads to muscle degeneration. Most forms of MD start in early childhood. in the group of disorders, dystrophinopathies are the most common. These include, Duchenne Muscular Dystrophy otherwise known as DMD and Becker Muscular Dystrophy, BMD. Both of these result in the mutation of the Dystrophin gene, in addition to DMD and BMD, genetic mutations in other genes are responsible for several other Muscular Dystrophies. The dystrophin gene is a major gene on the x chromosome, resulting in MD being more common I males as they only have one copy of the dystrophin gene while females have two, effectively leading to 1 out of every 3500 males being born with MD (Stefan Kochanek, 1995). Early Symptoms that may be seen in some Muscular Dystrophies are; pain and stiffness in the muscles, difficulty with running and jumping, walking on toes, difficulty sitting up or standing, learning disabilities, such as developing speech later than usual, and frequent falling. Some of the later symptoms that are more likely when the person has had the disease for a longer period of time may be; the inability to walk, the shortening of muscles and tendons limiting movement, breathing difficulty, and in some circumstances breathing can get so severe, assisted breathing may be necessary, and also difficulty swallowing with a risk of aspiration pneumonia (Tim Newman, 2017). Currently there are no treatments to cure Muscular Dystrophy, however, there are medications to help reduce pain, and special aids such as therapy and surgeries to help maintain some function of the muscle for longer. While many of the different MDs have vastly different symptoms, most of them have quite short life expectancies. For Duchenne Muscular Dystrophy, the life expectancy can be as short as early

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