Viral gene therapy as treatment for cholesterol storage disease or disorder
AssigneeThe United States of America, as represented by the Secretary, Department of Health and Human Servi
InventorsCharles P. Venditti, Randy Chandler, William J. Pavan
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Abstract
Provided herein are compositions and methods for the viral gene therapy (e.g., AAV-directed gene therapy) of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.
Claims15
A nucleic acid construct comprising a viral vector comprising (a) a mini-elongation factor 1 α (miniEF1α) promoter and (b) an NPC1 gene sequence operably linked to the miniEF1α promoter.
A nucleic acid construct comprising an adeno-associated virus (AAV) viral vector comprising (a) a first AAV2 inverted terminal repeat (ITR) having exactly 130 base pairs, (b) a mini-elongation factor 1 α (miniEF1α) promoter, (c) an NPC1 gene sequence operably linked to the miniEF1α promoter, (d) a rabbit beta globin poly A signal, and (e) a second AAV2 ITR having exactly 130 base pairs.
Dependencies are inferred from the claim text.