Investigators have safely injected gamma-sarcoglycan genes into an arm muscle in nine people with limb-girdle muscular dystrophy type 2C; high-dose recipients produced gamma-sarcoglycan protein

posted on March 14, 2012 - 9:24am
A March 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, presents the results of a phase 1 trial of gene therapy for gamma-sarcoglycan-deficient limb-girdle muscular dystrophy (LGMD), also known as LGMD2C.

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