Model construction of Niemann-Pick type C disease in zebrafish

Abstract excerpt

Niemann-Pick type C disease (NPC) is a rare human disease, with limited effective treatment options. Most cases of NPC disease are associated with inactivating mutations of the NPC1 gene. However, cellular and molecular mechanisms responsible for the NPC1 pathogenesis remain poorly defined. This is partly due to the lack of a suitable animal model to monitor the disease progression. In this study, we used CRISPR...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Autoimmune and Inflammatory Disorders Research
• Share to Cytomegalovirus and herpesvirus research
• Share to Lysosomal Storage Disorders Research