Gene therapy cures severe pediatric neurodegenerative disease in animal models

The following is an excerpt.

A single session of a gene therapy developed by the Universitat Autònoma de Barcelona (UAB) 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.

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Read the full story here: Gene Therapy Cures a Severe Pediatric Neurodegenerative Disease in Animal Models