Patents Assigned to Hope for Annabel
  • Patent number: 11738093
    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