Licensing Processes for Gene Therapy Products: Approved and Pending Clinical Trials In The World and Turkey


Sılan C., Güngör B.

Gene Therapy, AKPOLAT NEZAHAT, HASPOLAT YUSUF KENAN, Editör, NOBEL TIP KİTABEVLERİ, İstanbul, ss.153-187, 2024

  • Yayın Türü: Kitapta Bölüm / Mesleki Kitap
  • Basım Tarihi: 2024
  • Yayınevi: NOBEL TIP KİTABEVLERİ
  • Basıldığı Şehir: İstanbul
  • Sayfa Sayıları: ss.153-187
  • Editörler: AKPOLAT NEZAHAT, HASPOLAT YUSUF KENAN, Editör
  • Çanakkale Onsekiz Mart Üniversitesi Adresli: Evet

Özet

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.