Gene Therapy Cures a Severe Pediatric Neurodegenerative Disease in Animal Models

Gene Therapy Cures a Severe Pediatric Neurodegenerative Disease in Animal Models. Universitat Autònoma de Barcelona (2013, July 2). ScienceDaily. Retrieved July 3, 2013, from http://www.sciencedaily.com­ /releases/2013/07/130702100344.htm

July 2, 2013 — 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.


Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. Virginia Haurigot, Sara Marcó, Albert Ribera, Miguel Garcia, Albert Ruzo, Pilar Villacampa, Eduard Ayuso, Sònia Añor, Anna Andaluz, Mercedes Pineda, Gemma García-Fructuoso, Maria Molas, Luca Maggioni, Sergio Muñoz, Sandra Motas, Jesús Ruberte, Federico Mingozzi, Martí Pumarola, Fatima Bosch
J Clin Invest. 2013; doi:10.1172/JCI66778

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