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© Newswise.
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Gene Repair for Muscular Dystrophy
Gene repair for muscular dystrophy A type of gene therapy has been shown to provide long-term treatment for a form of muscular dystrophy (MD)--at least in dogs. Researchers tested the method using the golden retriever dog, a model organism for study of Duchenne MD, the most common childhood form of MD in humans. Gene therapy for MD has been more difficult up until now because the gene is so large it can't be delivered via more traditional means. The new method shows evidence for long-term repair of a specific genetic defect in a live animal using a chimeric oligonucleotide--a manufactured molecule that contains both DNA and RNA. Duchenne MD is caused by a defect in the gene that codes for dystrophin protein, which attaches to other proteins in muscle cells and helps anchor muscles to connective tissues. Scientists have devoted considerable effort to the study of gene therapy in the mouse model for MD--using methods such as direct injection of DNA, viral vectors, and transplantation of myoblasts (muscle cells)--but have had only modest short-term success because of problems with gene transfer or adverse immune responses. In the canine model of the disease, a mutation in mRNA processing leads to a deletion of part of the mRNA, resulting in the premature termination of the dystrophin protein. In the current experiment, led by Richard Bartlett, the chimeric oligonucleotide incorporated the corrected sequence into the chromosome using the cell's own DNA repair enzymes, resulting in the replacement of the aberrant RNA processing signal from the mRNA, and the restoration in production of the normal full-length protein. While the results are impressive, the technique still has a long way to go before its reaches the efficiencies required for use as a human therapeutic. ------------------------------------------------------------- Please fax or e-mail this form to Lynn Bhajan at l.bhajan@natureny.com>
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