Back to results
US 2018/0104289 A1Published application
Viral gene therapy as treatment for cholesterol storage disease or disorder
AssigneesThe United States of America, as represented by the Secretary, Department of Health and Human, Services
InventorsCharles P. Venditti, Randy Chandler, William J. Pavan
Life of this patent
Estimating the term…
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.
Claims25
A nucleic acid construct comprising:
- (1) a viral vector sequence;
- and
- (2) an NPC1 gene sequence under control of a mini-calmodulin promoter or a mini-elongation factor 1 α (miniEF1α) promoter.
(canceled)
(canceled)
A method for treating Niemann-Pick disease, Type C in a subject by gene therapy comprising administering a composition comprising a therapeutically effective amount of a gene therapy construct comprising (1) a viral vector sequence;
- and (2) an NPC1 gene sequence under control of a mini-calmodulin promoter or a mini elongation factor 1 α (miniEF1α) promoter, and a pharmaceutically acceptable carrier.
(canceled)
(canceled)
(canceled)
Dependencies are inferred from the claim text.