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 ...
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Cyclo Therapeutics reports promising NPC1 treatment findingsThe TransportNPC™ study is considered the most comprehensive of its kind concerning patient size, global reach, duration, and clinical outcomes for NPC1 treatment. The stock has shown resilience ...
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 ...
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