Translational Benefits of Gene Therapy to Date

Abstract

Gene therapy is now a reality with a number of early phase clinical trials having been completed and several currently in progress. In spite of some early setbacks substantial progress has been made with treatment of several different diseases using a variety of delivery vectors and transgenes. Indeed for some diseases gene therapy is now the treatment of choice, in particular the inherited immune deficiencies. Treatment of ocular diseases and cancer are also showing great promise. Immune responses and insertional mutagenesis still pose problems but refi nement of delivery systems and an increased understanding of oncogene activation should ensure that more successful protocols will emerge in the near future. Continuous progress suggests that a wider range of diseases can be treated with gene therapy in the future.

Keywords

• gene therapy
• neoplasm
• clinical trial
• immunology