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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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Detalles Bibliográficos
Autores principales:	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:	Online Artículo Texto
Lenguaje:	English
Publicado:	The Company of Biologists Ltd 2018
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6176986/ https://www.ncbi.nlm.nih.gov/pubmed/30135069 http://dx.doi.org/10.1242/dmm.034165

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