Area
Endocrine, Nutritional and Metabolic DiseasesIndication
Niemann-Pick Disease CSystem
In vitroModel references
JIMD Rep. 2012;4:29-37;
Cell Rep. 2014 May 8;7(3):883-97;
Hum Mutat. 2009 Jul; 30(7): 1117–1122
Niemann-Pick type C (NPC) mutant cell lines and patient fibroblasts
Applications
- New therapeutic strategies.
- Construction of transient and stable NPC gene silencing model.
- Functional studies.
