Gen tedavisinin temel ilkeleri ve son gelişmeler

Abstract

Gene therapy generally can be defined as the transfer of genetic material in cells with the aim of treating diseases or at least improving the patient's clinical status. The basic aim of gene therapy is to transfer the therapeutic gene into target cells using a vector. The most widely used gene therapy vectors are viral vectors, like adeno-associated virus, retrovirus, adenovirus and herpesvirus vectors. Gene therapy can be applied to both somatic and germline cells. The first gene therapy study, considered as a clinical application, was performed in 1982. Because of the extensive developments in gene therapy, it has attracted so many scientists to this field faster than expected. The advances in this field have provided the first gene therapy products, for example, approved gene therapy drugs for cancer and lipoprotein lipase deficiency treatment can be mentioned. In addition, gene therapy drugs for patients with Leber's congenital amaurosis (LCA) and hemophilia is currently in phase III study. In this review, the basic principles, history and current clinical applications of gene therapy are presented with up to date literature.