GENE THERAPY FOR ARRHYTHMOGENIC RIGHT VENTRICULAR CARDIOMYOPATHY

Compositions and methods for preventing and treating cardiac arrhythmia. Methods of preventing or treating arrhythmogenic right ventricular cardiomyopathy (ARVC), comprising administering to a subject in need a prophylactic or treatment effective amount of a composition comprising a plakophilin-2 (P...

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Bibliographic Details
Main Authors SHEIKH, Farah, BRADFORD, William
Format Patent
LanguageEnglish
French
German
Published 21.02.2024
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Summary:Compositions and methods for preventing and treating cardiac arrhythmia. Methods of preventing or treating arrhythmogenic right ventricular cardiomyopathy (ARVC), comprising administering to a subject in need a prophylactic or treatment effective amount of a composition comprising a plakophilin-2 (PKP2) gene. The composition further comprises an adenovirus-associated vector (AAV) to deliver the PKP-2 gene. In embodiments, the invention provides that the AAV is a cardiotropic AAV serotype and contains a cardiac-specific promoter. Method of treating a cardiovascular disease characterized by abnormal cardiac cell-cell junction complex comprising administering to a subject in need a prophylactic or treatment effective amount of a composition comprising a plakophilin-2 (PKP2) gene.
Bibliography:Application Number: EP20220788797