Duchenne Disorder Research Paper

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Biology Research Paper Duchenne Disorder (Duchenne Muscular Dystrophy) Duchenne disorder is classified as a very severe, rare case of muscular dystrophy, which is a group of inherited disorders that cause muscle weakness. It affects all voluntary muscles, as well as the heart and breathing muscles. This particular disease is, in fact, genetic. Unlike a lot of genetic disorders, Duchenne disorder occurs in people that do not have a family history of the condition. According to a study by the Muscular Dystrophy Campaign, in the United Kingdom, 1 of every 3500 males is born with Duchenne disorder. It obviously occurs by a mutation on a genetic code. It is found to be caused by a defective gene for dystrophin, which is a protein within the…show more content…
Because of the way the disease is inherited, it is much more likely for a male to obtain the disease because they have only one X chromosome, whereas females have two so it would be more difficult for them to receive it on both of their chromosomes. According to the Muscular Dystrophy Campaign, more than half of the cases of Duchenne disorder are inherited from the mother. This is because it is likely for a mother to be a carrier by the gene, but they are not typically affected by the disorder. Looking at it that way, the son of a carrier has a fifty-fifty chance of being affected while the daughter has a fifty-fifty chance of being a carrier alone. According to the same source, quite a small amount of female carriers experience a very small degree of muscle weakness…show more content…
According to science daily, scientists have been working on a cure for this type of muscular dystrophy and it is known as exon skipping, which is currently in clinical trial for Duchenne muscular dystrophy. This phenomenon involves short strands of synthetic DNA that are essentially used as a sort of molecular patch. This patch restores the production of the protein dystrophin, which ends up hiding the faulty piece of the dystrophin gene and allows a functional protein to be produced. The treatment will be injected intramuscular. Scientists believe this method will transform the severe disease of Duchenne muscular dystrophy to a much milder Becker muscular dystrophy. For me, some pros to this approach would be a cure for a severe case of muscular dystrophy, but it is unfortunate that the patient will still have the disorder, just in a milder form. A con would be any side effects that exon skipping may have. Since there are not any recorded cases of this being done in the United States, it is hard to know whether or not exon skipping could be potentially dangerous to those who want to try the treatment since testing in human patients is still out of the question. Another con is within the administration of the treatment. Since Duchenne muscular dystrophy affects every single voluntary muscle, there is a

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