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Reserch And Treatment Of Muscular Dystrophy Disease

653 words - 3 pages

Muscular dystrophy is a disease characterized by the weakness and deterioration of skeletal muscles. There are over 30 different forms of muscular dystrophy but the two most common are Duchenne and Becker Muscular Dystrophy (Baroncelli). These two forms of the disease are sex linked, affecting primarily males. A male usually inherits a faulty X chromosome from his mother and receives no other X chromosome to offset the faulty one (males are XY). Affected persons have difficulty performing daily tasks and eventually lose the ability to walk. However, a faulty X chromosome inherited by a female can be offset by a normal X chromosome inherited from the other parent (females are XX). ...view middle of the document...

Ataluren is a drug that uses ribosomes to read through premature stop signals of mRNA, which allows the cell to produce a functional protein (ptcbio). In the case of muscular dystrophy, patients have the potential to delay muscular degeneration provided their immune systems accept the drug. Although muscular dystrophy is not curable at this moment, Ataluren gives the hope of prolonging the patient’s muscle system.

A trial conducted by Mendell (1995) was done using myoblasts as the point of focus in tissue regeneration. The myoblasts were to be injected into patient’s arms with Duchenne’s muscular dystrophy. The hope was for the myoblasts to fuse with muscle fibers and contribute their nucleus, thus creating a functional gene to produce efficient proteins. The 12 participants in the trials did not have any significant change in muscle strength yielding unsuccessful results. A more promising experiment conducted on mice has shown the regeneration of damaged muscle tissue fibers using bone marrow. The growth and repair of damaged skeletal muscles are caused by the absence of satellite...

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