Gene Therapy: A Newfangled Technology for Genetic Modification

Abstract

Gene therapy provides modern medicine with new perspectives that were unthinkable two decades ago and it has been proposed as a future strategy to prevent aging. Gene therapy is “the use of genes as medicine” involving the transfer of a therapeutic or working copy of a gene into specific cells of an individual in order to repair a faulty gene copy. Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by use of recombinant viruses (sometimes called biological nanoparticles or viral vectors) or those that use naked DNA or DNA complexes (non-viral methods). Although gene therapy as a treatment for disease holds great promise, progress in developing effective clinical protocols has been slow. The problem lies in the development of safe and efficient gene-delivery systems. This review summarizes various methods, vectors and gene delivery techniques of gene therapy including current status and recent developments in gene therapy.

Keywords: Gene therapy, viral vectors, non-viral vectors, gene delivery systems, ethical issues

Gene therapy provides modern medicine with new perspectives that were unthinkable two decades ago and it has been proposed as a future strategy to prevent aging. Gene therapy is “the use of genes as medicine” involving the transfer of a therapeutic or working copy of a gene into specific cells of an individual in order to repair a faulty gene copy. Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by use of recombinant viruses (sometimes called biological nanoparticles or viral vectors) or those that use naked DNA or DNA complexes (non-viral methods). Although gene therapy as a treatment for disease holds great promise, progress in developing effective clinical protocols has been slow. The problem lies in the development of safe and efficient gene-delivery systems. This review summarizes various methods, vectors and gene delivery techniques of gene therapy including current status and recent developments in gene therapy.

Keywords: Gene therapy, viral vectors, non-viral vectors, gene delivery systems, ethical issues