NPC1 trafficking via VPS41-dependent LAMP carriers regulates endosomal cholesterol homeostasis. [PDF]
Ndoj K +10 more
europepmc +1 more source
Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease [PDF]
et al,, Jiang, Xuntian, Ory, Daniel S
core +1 more source
Early Diagnosis of Nieman-Pick Disease Type C and Rapid Response of Gelastic Cataplexy to Treatment With N-Acetyl-L-Leucine: A Case Report. [PDF]
Rojnueangnit K +3 more
europepmc +1 more source
Comparison of neonatal systemic and intracerebroventricular AAV9 gene therapy delivery demonstrating improved behavioral and phenotypic outcomes in a mouse model of Niemann-Pick disease, type C1. [PDF]
Epstein BE +6 more
europepmc +1 more source
Targeting Pathways Implicated in Cholesterol Metabolism for Novel Cancer Therapy. [PDF]
Zhou Y +15 more
europepmc +1 more source
Ligand Design Criteria for the Stability of High Oxidation State Praseodymium Complexes. [PDF]
Nero TR +5 more
europepmc +1 more source
A clickable CoQ imaging probe reveals that cellular uptake and lysosomal trafficking depend on CD36 and NPC1. [PDF]
Liparulo I +8 more
europepmc +1 more source
Characterization of liver disease in a cohort of individuals with Niemann-Pick Disease, Type C1. [PDF]
Agrawal N +8 more
europepmc +1 more source

