Medical Xpress8d
Assessing the prevalence of neutralizing antibodies in individuals with Niemann-Pick disease
NPC1 is a rare, fatal neurodegenerative disorder, for which systemic AAV9-based gene therapy in mouse models has shown some success. The presence of neutralizing antibodies against AAV can reduce ...
Medscape15d
NPC1 in Human White Adipose Tissue and Obesity
Background: Genetic studies have implicated the NPC1 gene (Niemann Pick type C1) in susceptibility to obesity. Methods: To assess the potential function of NPC1 in obesity, we determined its ...
eLife19d
Small-molecule Activation of TFEB Alleviates Niemann-Pick Disease Type C via Promoting Lysosomal Exocytosis and Biogenesis
TFEB overexpression or pharmacological activation of TFEB ameliorates cholesterol accumulation in U18666A-induced HeLa NPC1 model. A working scheme to illustrate that small-molecule TFEB agonist ...
eLife19d
Small-molecule Activation of TFEB Alleviates Niemann-Pick Disease Type C via Promoting Lysosomal Exocytosis and Biogenesis
Here, we show that transgenic overexpression of TFEB, but not TFE3 (another member of MiTF family) facilitates cholesterol clearance in various NPC1 cell models. Pharmacological activation of TFEB by ...
Frontiers14d
Baculovirus Utilizes Cholesterol Transporter NIEMANN–Pick C1 for Host Cell Entry
BmE cells (2 × 10 5 cells/well) seeded in twelve-well culture plates were incubated with one of the following antibodies: mouse anti-NPC1-A, anti-NPC1-I polyclonal antibody, rabbit anti-NPC1-C ...