The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Aug. 29, 2023
Filed:
Apr. 09, 2019
Cure Ahc, Inc., Raleigh, NC (US);
Hope for Annabel, Washington, DC (US);
Alternating Hemiplegia of Childhood Foundation, Southfield, MI (US);
Simon Frost, Washington, DC (US);
Natalia Morsci, Salt Lake City, UT (US);
Neil Hackett, Santa Monica, CA (US);
Dolan Sondhi, New York, NY (US);
Hope for Annabel, Washington, DC (US);
Cure AHC, Inc., Raleigh, NC (US);
Alternating Hemiplegia of Childhood Foundation, Southfield, MI (US);
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.