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US 12,201,658 B2Granted
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 Services
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.
Claims13
A method for treating or preventing a cholesterol storage disease or disorder in a subject, the method comprising:
- administering a composition comprising (1) a therapeutically effective amount of 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, and (2) a pharmaceutically acceptable carrier to the subject,
- wherein the cholesterol storage disease or disorder is caused by mutation or malfunction in the NPC1 gene of the subject,
- thereby treating or preventing the cholesterol storage disease or disorder in the subject.
A method for treating or preventing a cholesterol storage disease or disorder in a subject, the method comprising:
- administering a composition comprising (1) a therapeutically effective amount of 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, and (2) a pharmaceutically acceptable carrier to the subject,
- wherein the cholesterol storage disease or disorder is caused by mutation or malfunction in the NPC1 gene of the subject,
- thereby treating or preventing the cholesterol storage disease or disorder in the subject.
Dependencies are inferred from the claim text.