Chapter 16 - Gene and protein-based therapies for improving cardiac performance and regeneration

Abstract

Gene-based therapy is a relatively novel treatment which has the ability to target intracellular disease-specific manifestations in the setting of cardiac dysfunction. Gene transfer offers an option to treat the mutated or failing cardiomyocytes via introducing genetic material into the cells. Achieving efficient cardiac gene therapy requires the application and interaction of several essential factors including: the selection of an appropriate cardiac-tropic vector and promoter, creation of effective modes of gene transfer, and validation of the correct transgene molecular targets. In this chapter we survey gene and protein-based approaches for the enhancement of cardiac function in heart failure and improvement of right ventricular function in pulmonary arterial hypertension, and discuss emerging concepts and innovation in cardiac regeneration.