Article
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice.
Life science alliance - 1 Oct 2021
Davidson Cristin D, Gibson Alana L, Gu Tansy, Baxter Laura L, Deverman Benjamin E, Beadle Keith, Incao Arturo A, Rodriguez-Gil Jorge L, Fujiwara Hideji, Jiang Xuntian, Chandler Randy J, Ory Daniel S, Gradinaru Viviana, Venditti Charles P, Pavan William J
Abstract excerpt
Niemann-Pick C1 disease (NPC1) is a rare, fatal neurodegenerative disease caused by mutations in NPC1, which encodes the lysosomal cholesterol transport protein NPC1. Disease pathology involves lysosomal accumulation of cholesterol and lipids, leading to neurological and visceral complications. Targeting the central nervous system (CNS) from systemic circulation complicates treatment of neurological diseases with...
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