Plakophillin-2 gene therapy methods and compositions
AssigneeTenaya Therapeutics, Inc.
InventorsZhihong Jane YANG, Jaclyn HO, Chris REID, Jin YANG
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Abstract
Provided herein are methods and compositions for plakophilin-2 gene therapy for treating heart diseases such as arrhythmogenic right ventricular cardiomyopathy (ARVC) or arrhythmogenic cardiomyopathy (ACM).
Claims19
A method for restoring desmosome structure and/or function in an individual in need thereof, the method comprising administering an adeno-associated virus (AAV) gene therapy vector comprising a nucleic acid encoding a plakophilin 2 (PKP2) polypeptide operatively linked to a promoter, wherein the AAV is selected from the group consisting of an AAV6, an AAV8, an AAV.rh74, and an AAV9, thereby restoring desmosome structure and/or function in the individual.
Dependencies are inferred from the claim text.