Abstract: This invention relates to a transgenic vector for transducing cells of a mammal's CNS and transgenically expressing a protein in the mammal's CNS. The transgenic vector comprises a virus-derived vector, a nucleic acid sequence encoding the protein, and an endogenous ATP1A3 promoter sequence. This invention also relates to a composition comprising a recombinant AAV vector comprising a nucleic acid sequence encoding a ATP1A3 protein, in a form compatible with administration into the CNS. The invention also relates to a method for treating a subject having a neurological disorder associated with mutations in the ATP1A3 gene and a method for delivering a transgenic ATP1A3 DNA to the central nervous system of a mammal by administering the recombinant AAV vector into the mammal's CNS.
Type:
Grant
Filed:
April 9, 2019
Date of Patent:
August 29, 2023
Assignees:
Hope for Annabel, Cure AHC, Inc., Alternating Hemiplegia of Childhood Foundation
Inventors:
Simon Frost, Natalia Morsci, Neil Hackett, Dolan Sondhi