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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Dades bibliogràfiques
Publicat a:Hum Gene Ther
Autors principals: Kurokawa, Yoshie, Osaka, Hitoshi, Kouga, Takeshi, Jimbo, Eriko, Muramatsu, Kazuhiro, Nakamura, Sachie, Takayanagi, Yuki, Onaka, Tatsushi, Muramatsu, Shin-ichi, Yamagata, Takanori
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc., publishers 2021
Matèries:
Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8236559/
https://ncbi.nlm.nih.gov/pubmed/33256498
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2020.175
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