Gene Therapy for Cardiac Transplantation

Abstract

Gene therapy is an advanced treatment approach that alters the genetic composition of cells to confer therapeutic protein or RNA expression to the target organ. It has been successfully introduced into clinical practice for the treatment of various diseases. Cardiac transplantation stands to benefit from applications of gene therapy to prevent the onset of post-transplantation complications, such as primary graft dysfunction, cardiac allograft vasculopathy, and rejection. Additionally, gene therapy can be used to minimize or potentially eliminate the need for immunosuppression post-transplantation. Several animal models and delivery strategies have been developed over the years with the goal of achieving robust gene expression in the heart. However, a method for doing this has yet to be successfully translated into clinical practice. The recent advances in ex vivo perfusion for organ preservation provide potential ways to overcome several barriers to achieving gene therapy for cardiac transplantation into clinical practice. Optimizing the selection of the gene-carrying vector for gene delivery and selection of the therapeutic gene to be conferred is also crucial for being able to implement gene therapy in cardiac transplantation. Here, we discuss the history and current state of research on gene therapy for cardiac transplantation.