Abstract

Gene Therapy, Past, Present, and Future

Masafumi Onodera, Head, Department of Human Genetics, National Center for Child Health and Development

Almost 30 years ago, the first gene therapy clinical trial was conducted for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SICD) in the US and Italy. Since then, the large number of gene therapy have been done and made steady progress despite of swings and roundabouts and currently proven to be a safe and effective therapeutic option for various intractable diseases such as primary immunodeficiencies, inherited metabolic disorders, hemoglobinopathy, inherited retinitis, hemophilia A and B, neuromuscular diseases, hematological malignancy, and some solid tumors. Furthermore, a new technology using genome editing for the diseases has been developed in rapid pace and applicable in clinical trials and then the definition of gene therapy may change from “therapy with a gene” to “therapy for a gene” in near future. Considering what coming from such a new technology sometimes carry unimaginable risks, however, the discussion from the wide angle including an ethical point of view should be needed to put into practice the new technology.  From the point of this view, we have to ask ourselves “What can we do and What should we do” to future gene therapy. I would like to think and discuss the best course of action to the progress of gene therapy together in my presentation.