Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

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. Targ...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Life Science Alliance LLC 2021
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8380657/
https://www.ncbi.nlm.nih.gov/pubmed/34407999
http://dx.doi.org/10.26508/lsa.202101040

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8380657/
https://www.ncbi.nlm.nih.gov/pubmed/34407999
http://dx.doi.org/10.26508/lsa.202101040

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