Gene Therapy Clinical Trials: Past, Present and Future

Abstract This chapter gives an overview of the gene therapy clinical trials through the years, from the first attempts to deliver a correct DNA sequence to cells of a patient suffering from a genetic disease to the most recent trials where more complex or more targeted gene therapy approaches have been carried out. The types of vectors used, some of the clinical problems linked to them, cases of frustrating failures or severe adverse events are described together with many clinical successes. The history of gene therapy clinical trials shows light and dark but in the end gene therapy has been proven to provide durable benefits to patients. Eventually some of the investigated gene therapy medicinal products gained market approval both in the United States and the European Union. Gene therapy is increasingly becoming an important tool in the medical armamentarium for patient treatment.