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Gene Therapy in a Mouse Model of Niemann–Pick Disease Type C1

Niemann–Pick disease type C1 (NPC1) is a fatal congenital neurodegenerative disorder caused by mutations in the NPC1 gene, which is involved in cholesterol transport in lysosomes. Broad clinical manifestations of NPC1 include liver failure, pulmonary disorder, neurological deficits, and psychiatric...

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Detalles Bibliográficos
Autores principales:	Kurokawa, Yoshie, Osaka, Hitoshi, Kouga, Takeshi, Jimbo, Eriko, Muramatsu, Kazuhiro, Nakamura, Sachie, Takayanagi, Yuki, Onaka, Tatsushi, Muramatsu, Shin-ichi, Yamagata, Takanori
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2021
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8236559/ https://www.ncbi.nlm.nih.gov/pubmed/33256498 http://dx.doi.org/10.1089/hum.2020.175

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