Gene therapy cures a severe pediatric neurodegenerative disease in animal models
Tuesday, July 2, 2013 - 16:30
in Health & Medicine
A single session of gene therapy cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects between 1 and 9 out of every 100,000 children, and causes the death of the child on reaching adolescence.