Innovative Gene Therapy for Heart Disease: p21 Overexpression in Cardiomyocytes

University of Pittsburgh researchers have developed a viral vector that allows for the overexpression of Cdkn1a cDNA selectively in cardiomyocytes. This innovation increases cardiomyocyte p21 protein levels, which has been shown to decrease pathological growth in the heart in preclinical murine models of hypertrophic cardiomyopathy. Additionally, this treatment improves diastolic function, offering a promising new approach to managing this genetic heart condition.

Description

The invention utilizes an AAV9 vector to selectively increase p21 protein levels in cardiomyocytes. This targeted gene therapy approach reduces pathological remodeling in genetic forms of hypertrophic cardiomyopathy, a condition characterized by abnormal thickening of the heart muscle. By enhancing p21 expression, the therapy mitigates the adverse effects of the disease and improves heart function.

Applications

- Gene therapy for hypertrophic cardiomyopathy
- Treatment of cardiovascular diseases
- Research tool for studying cardiac hypertrophy and regeneration

Advantages

The use of an AAV9 vector ensures that the overexpression of p21 is specific to cardiomyocytes, minimizing off-target effects. Preclinical studies have demonstrated that this therapy not only reduces pathological growth but also enhances diastolic function. This method represents a unique strategy for addressing hypertrophic cardiomyopathy by leveraging the body's own cellular mechanisms to counteract disease progression.

Invention Readiness

The technology has been validated in vivo, with data showing significant therapeutic effects in preclinical murine models. Experimental results indicate that the viral vector successfully increases p21 protein levels in cardiomyocytes, leading to a reduction in pathological heart growth and improvement in diastolic function. These findings suggest a strong potential for further research and eventual clinical application, with ongoing studies to optimize dosage and delivery methods.

IP Status

Patent pending