Gene Therapy for the Heart

Abstract

The focus of this chapter is genetic engineering and gene therapy. We begin by introducing the genetic engineering paradigm and then move on to provide specific examples describing the successful application of genetic engineering technology. Next, we discuss some of the important milestones in the field of genetic engineering. Recent advances in the field of genetic engineering have resulted in the evolution of a completely new concept—personalized medicine, which is described next. The next three sections are focused on recombinant DNA technology, starting with an overview of the human genetic code, followed by a description of plasmid vectors and ending by discussing the process related to recombinant DNA technology. The next four sections are related to concepts in cardiac gene therapy. We begin by introducing the concept of cardiac gene therapy and present a three-step process outlining cardiac gene therapy. We then describe different strategies that have been used for vector delivery to the heart and also discuss SERCA2a as a potential therapeutic target for cardiac gene therapy. After this, we describe many of the different viral vectors used in gene therapy. We end this chapter by presenting two case studies, one that describes the use of genetic engineering for reprogramming of adult somatic cells to form contractile cardiac cells and the second one that makes use of genetic engineering technology to improve vascularization in 3D tissue.