Gene Therapy Trial Yields Promising Results

Promising study results for LGMD2D gene therapy trial:  Dr. Mendell injected alpha sarcoglycan using AAV vector into muscle of LGMD mouse model.  

Two promoters, muscle creatine kinase (MCK) and truncated MCK, led to sustained production of alpha-sarcoglycan protein in 60 percent to 70 percent of the fibers in the injected area six and then 12 weeks after the injections.  Neurology.2008 July 22;71 (4):240-7

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    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...

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