Gene Therapy, AKPOLAT NEZAHAT, HASPOLAT YUSUF KENAN, Editör, NOBEL TIP KİTABEVLERİ, İstanbul, ss.153-187, 2024
Gene therapy represents one of the most important and exciting are as of modern medicine. Basically, gene therapy is medical technology
aimed at treating diseases through the alteration, insertion or correction of genes (1). This method is designed to get to the root cause of
diseases and address genetic defects. The basic principles of gene therapy are the correct transportation of genetic information, its delivery
to targeted cells and its effective application. These basic principles are key to the success of gene therapy. Gene therapy offers potential solu-
tions for the treatment of many genetic diseases. Gene therapy can play an important role in the treatment of genetic-based diseases such as
cystic fibrosis, blood cancers and neurological disorders. Furthermore, gene therapy could form the basis of future medical treatments.
However, the clinical trials and approval process in this field require rigorous conduct and careful evaluation. In this section, the important
stages of gene therapy products from pre-clinical research to human trials and the approval process will be discussed, followed by approved
products and products in phase studies.