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Niemann Pick type C disease (NP-C) is a neurodegenerative lysosomal storage disorder caused, in most cases, by mutations in the NPC1 gene. This leads to the accumulation of cholesterol and other lipids in various cells of the body. However, the subsequent premature death is usually associated to the neurological manifestations. Advances in viral vector technology and recent successful clinical trials have meant that gene therapy may have the potential to treat NP-C. To e...
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