|First clinical trial of gene therapy for muscular dystrophy lends insight into the disease|
|Thursday, October 07, 2010|
A clinical trial designed to replace the genetic defect causing the most common form of muscular dystrophy has uncovered an unexpected aspect of the disease. The trial, based on therapy designed by scientists at the University of North Carolina at Chapel Hill School of Medicine, showed that some patients mount an immune response to the dystrophin protein even before they have received the gene therapy.
The puzzling results, which came from trials at Columbus Children’s Hospital in Ohio, suggest that the immune systems of a number of patients -- once thought to be completely devoid of the dystrophin protein -- are actually primed by the prior existence of tiny amounts of this important component of muscle.
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