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Duchennes muscular dystrophy is the result of a defective gene on the Xchromosome This gene is responsible for production of the muscle protein dystrophin Dystrophin is an integral part of the dystrophin-glycoprotein complex which bears thebrunt of the force generated during muscular contraction When dystrophin is notproduced the dystrophin-glycoprotein complex DCG is not present Absence of theDCG leads to tears in the muscle membrane because the muscle membrane bears the forceof muscular contraction alone Tears in the muscle membrane allow substances to leak inand out of the muscle fibers at random This uncontrolled biochemical traffic leads toeventual death of the muscle fibers Most of the current research on Duchennes muscular dystrophy involves genetherapy Researchers are attempting to find ways to introduce a healthy dystrophin geneinto the afflicted individual This healthy gene would produce the dystrophin proteinthereby regenerating the DGC which would in turn curb muscle fiber death Studies withmice have shown that introduction of the dystrophin gene is effective in treatingDuchennes muscular dystrophy However introduction of the dystrophin gene into thebody is no easy task Thus many scientists are focusing their research on ways to presentViruses have a natural inclination to deposit their genetic material in a cellsnucleus and thus are primary candidates for gene transport The dystrophin gene is arelatively large gene and therefore must be delivered via an adenovirus The problem withviral delivery is that the immune system of the recipient recognizes the virus as foreign anddestroys both the virus and the protein it is carrying Researchers at the University ofMichigan-Ann Arbor have developed an adenvirus that is gutted of its own geneticmaterial and consists only of a viral shell These gutted adenoviruses elicit fewerimmune responses However it is believed that immunosuppressant drugs such as FK506may be necessary to fully overcome the immune response to adenovirus-based geneAll current gene based research has been performed on animals but this fallinvestigators at the University of Ohio-Columbus and the University of Michigan-AnnArbor will begin a very
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