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
Published 12.10.2023
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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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: AU20220258329