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Published on Jan 16, 2012
http://www.telethon.it/en - Metachromatic leukodystrophy is a rare metabolic disease of genetic origin that affects the nervous system. Children are apparently normal at birth but then progressively lose all cognitive and motor faculties. The diseases mechanisms and research prospects are explained by Alessandra Biffi and Maria Sessa, researchers at the San Raffaele-Telethon Institute for gene therapy in Milan, who are carrying out the first clinical trials in the world to verify the safety and effectiveness of gene therapy for this very serious genetic disease.