CODON-OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF NIEMANN-PICK TYPE C1 DEFICIENCY AND RELATED CONDITIONS

摘要

The present disclosure provides compositions for viral gene therapy, e.g. Adeno-Associated virus-directed gene therapy, and methods of using the same for the treatment and/or prevention of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.