Chapters authored
Gene Therapy for Heart Disease: Modified mRNA Perspectives
Ischemic heart disease (IHD) presents a gigantic clinical challenge that demands effective therapeutic approaches. With increasing knowledge of the basic molecular mechanisms guiding the progress of this disease, it is now possible to target the key pathological players through gene therapy. Modified mRNA-based gene delivery presents a promising alternative to traditional gene therapy, because modRNA approaches have high potency, non-immunogenicity, greater efficiency and controlled nucleic acid transfer to the body. However, until recently the therapeutic applications of mRNA have been limited, as naturally occurring mRNA is rapidly degraded and cleared from the circulation. In this chapter, we outline the compositional changes made to mRNA to enhance its translational capacity and discuss the available carrier molecules currently being employed to deliver modRNA to the heart. We provide a detailed overview of modRNA applicability for cardiac repair and regeneration and consider future directions for novel delivery methods that can facilitate its cardiac therapeutic use.
Part of the book: Cardiomyopathy