A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease

Niemann–Pick disease type C1 (NP-C) is a prematurely lethal genetic lysosomal storage disorder with neurological and visceral pathology resulting from mutations in the NPC1 gene encoding the lysosomal transmembrane protein NPC1. There is currently no cure for NP-C, and the only disease modifying tre...

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Detalles Bibliográficos
Autores principales: Hughes, Michael Paul, Nelvagal, Hemanth Ramesh, Coombe-Tennant, Oliver, Smith, Dave, Smith, Claire, Massaro, Giulia, Poupon-Bejuit, Laura, Platt, Frances Mary, Rahim, Ahad Abdul
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10296851/
https://www.ncbi.nlm.nih.gov/pubmed/37371089
http://dx.doi.org/10.3390/cells12121619

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10296851/
https://www.ncbi.nlm.nih.gov/pubmed/37371089
http://dx.doi.org/10.3390/cells12121619

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