Muscular Dystrophy

Duchenne and Becker's MD are most common in children, and boys usually get the disease between the ages of two and 15 years of age (Duchenne, 2000). With Becker's MD it is possible to live a normal life, but with Duchenne MD, the life-span will be shortened. Scientists believe that the dystrophin gene on the X-chromosome is damaged in MD. The gene codes for the dystrophin protein, which is necessary for the structural support of muscles (Gregorevic et al., 2004). In MD, lack of dystrophin production leads to weakening of muscles, and in time the body stops making muscle cells and replaces them with fat and connective tissue.

Myotonic MD is the most common form of MD in adults and affects the eyes, heart, hormonal system, and blood, as well as causing muscular dystrophy and involuntary muscle sti

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stal muscles, as well as to cardiac myocytes, and gene expression persisted for at least 15 months after administration of the integrating vector. This study demonstrates the concept of in utero delivery for therapeutic and long-term prevention/correction of muscular dystrophy, and for other genetic disorders. Another group of researchers have found a method of gene delivery which reaches the muscles, of the heart, diaphragm and limbs and reverses the process of wasting found in muscular dystrophy (Gregorevic et al., 2004, 826-834). They have gone a step further than Gregory et al in producing the effects in an adult animal, and reversing the effects of MD already present. Gregorevic and his coworkers used a viral vector - a specific type of adeno-associated virus (AAV), which is able to home in on muscle cells and does not trigger a response by the immune system. The delivery system includes the use of vector growth factor, VEGF, which appears to increase penetration of the gene therapy vector into the muscles. Gregorevic et al say that by giving one injection of this AAV vector carrying a mini-dystrophin gene into the bloodstream, it is possible to deliver therapeutic levels of dystrophin to every skeletal and cardiac
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Approximate Word count = 1485
Approximate Pages = 6 (250 words per page)

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