Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions
AssigneeThe United States of America, as represented by the Secretary, Department of Health and Human Services
InventorsCharles P. Venditti, William J. Pavan, Randy J. Chandler
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Abstract
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.
Claims29
A nucleic acid construct for the expression of a therapeutic amount of NPC1 in a cell, the construct comprising a human codon-optimized NPC1 gene, optionally wherein:
- (i) the NPC1 is translationally fused to a protein transduction domain (PTD) to form a NPC1-PTD fusion protein, wherein the fusion protein is capable of cross-correcting non-transformed neighboring cells,
- (ii) the NPC1 gene is under the control of a promoter, and/or
- (iii) the NPC1 is translationally fused to a tag moiety.
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Dependencies are inferred from the claim text.