Gene therapy applications as treatment approach

Abstract

What is gene therapy? It is one of the treatment methods developed to be used in the treatment of hereditary diseases. It is based on a plan to rearrange faulty gene functions with gene therapy, which is still very new research, or to replace faulty genes with healthy ones through gene transfer. The gene therapy method was first introduced by Martine Cline in 1970. Martine Cline also suggested that viruses can be vectors that can be used to perform gene transfer. In 1980, he showed that genes can be transferred by ex vivo and in vivo methods. Then, in 1990, two children were fully cured as a result of gene therapy with a retrovirus vector carrying the ADA gene by Michael Blaese and William French Anderson in severe combined immunodeficiency (SCID) disease, and thus the first successful gene therapy procedure took place.