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US 2025/0049958 A1Published application
Gene therapy delivery of parkin mutants having increased activity to treat parkinson's disease
AssigneeNysnoBio GT Neurology, LLC
InventorJennifer JOHNSTON
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Abstract
The present disclosure provides novel gene therapy constructs comprising a PARK2 gene or activating variant and methods of administering to treat Parkinson's Disease or symptoms thereof.
Claims14
A composition comprising an AAV5 vector wherein the AAV5 vector comprises from 5′-3′:
- a. an AAV2 ITR;
- b. a CBA promoter;
- c. a wild-type PARK2 gene comprising a polynucleotide sequence that encodes SEQ ID NO:
- 2;
- d. a SV40 poly A tail;
- and
- e. an AAV2 ITR;
- wherein the AAV also comprises an AAV2 Rep and AAV5 cap genes.
5 more elements
Dependencies are inferred from the claim text.