Gene Therapy for Human Diseases: Recent Achievements and Near-Term Development Prospects

Abstract

The article briefly summarizes recent advances in genetic medicine that paved the way for the further development of gene therapy and set the stage for the development of next generation technology. Issues related to the main obstacle for wider application of gene therapy methods, in particular, with the immune response to gene delivery vectors and transgene products are considered. In this context, the role of new technology allowing to bypass the immune obstacle, such as development of modified capsids of adeno-associated viruses (AAV) and methods for temporary removal of antibodies from the bloodstream, as well as gene transfer into tissues using nanoparticles, is discussed. Along with the technology of the first generation gene therapy focused on the delivery of transgenes into target tissues, latest advances in the development of a completely new approach to gene therapy which is based on precise modification of the human genome sequence, gene editing technology, are summarized. Finally, promising next-generation gene editing technology is outlined, such as RNA-targeted editing technology and epigenome editing technology, which are more specific, precise, efficient and applicable to different groups of diseases. The article concludes that gene therapy and, in particular, human genome editing is perhaps the most exciting and revolutionary biotechnology of our time, due to both recent developments and opportunities it might provide in the nearest future.