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

• Main Authors: Wei-Chia Tseng (Author), Hannah E. Loeb (Author), Wuhong Pei (Author), Chon-Hw (Author), Lisha Xu (Author), Celine V. Cluzeau (Author), Christopher A. Wassif (Author), Benjamin Feldman (Author), Shawn M. Burgess (Author), William J. Pavan (Author), Forbes D. Porter (Author)
• Format: Book
• Published: The Company of Biologists, 2018-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...