Application Granted: 1/7/2025

Inventors:

Farah Sheikh Ph.D., CVS, FAHA, Professor of Department of Medicine UC San Diego; Jing Zhang Ph.D., Postdoctoral Fellow Researcher 

Connexin 43 (Cx43) is a cardiac ventricular gap junction protein crucial for cellular communication and cardiac function. Connexin 43's critical role in cardiac rhythms is exemplified in patients suffering from arrhythmogenic cardiomyopathy. Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) is a genetic heart muscle disease characterized by ventricular arrhythmias and structural abnormalities. Mutations in desmosomal genes, which are responsible for cell adhesion, are a major cause of ARVC. These mutations can lead to reduced expression and abnormal localization of Cx43.

This patent covers a gene therapy for ARVC, which consists of an adeno-associated virus (AAV) vector encoding the Cx43 protein operably linked to a promoter that is active in cardiac muscle tissue, allowing an increase in the expression of Cx43 protein levels in the heart. This gene therapy has been licensed and is in the preclinical stage.

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