Cure AHC, Inc.;Hope for Annabel;Alternating Hemiplegia of Childhood Foundation
发明人:
Simon Frost,Natalia Morsci,Neil Hackett,Dolan Sondhi
申请号：
US16379440
公开号：
US20190358346A1
申请日:
2019.04.09
申请国别(地区):
US
年份:
2019
代理人:
摘要：
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.
