Friday, January 13, 2012
Genethon, the Evry, France-based research lab created by the French Muscular Dystrophy Association, reported that promising results of a Phase I trial of gene therapy for limb-girdle muscular dystrophy type 2C have been published in Brain. The primary objective of the trial, which started in 2006, was to evaluate the safety of local injection of increasing doses of an adeno-associated virus vector harboring a "healthy" copy of the gene for gamma-sarcoglycan, the defective protein involved in the disease. Secondary objectives included assessing local and systemic immune reactions, as well as the quality of gene transfer in terms of efficacy, expression and distribution.
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