Modeling Niemann-Pick disease type C1 in zebrafish: a robust platform for in vivo screening of candidate therapeutic compounds

Niemann-Pick disease type C1 (NPC1) is a rare autosomal recessive lysosomal storage disease primarily caused by mutations in NPC1. NPC1 is characterized by abnormal accumulation of unesterified cholesterol and glycolipids in late endosomes and lysosomes. Common signs include neonatal jaundice, hepat...

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Detalhes bibliográficos
Publicado no:Dis Model Mech
Main Authors: Tseng, Wei-Chia, Loeb, Hannah E., Pei, Wuhong, Tsai-Morris, Chon-Hwa, Xu, Lisha, Cluzeau, Celine V., Wassif, Christopher A., Feldman, Benjamin, Burgess, Shawn M., Pavan, William J., Porter, Forbes D.
Formato: Artigo
Idioma:Inglês
Publicado em: The Company of Biologists Ltd 2018
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6176986/
https://ncbi.nlm.nih.gov/pubmed/30135069
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1242/dmm.034165
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