Gen tedavisinin temel ilkeleri ve son gelişmeler

Gen tedavisi, genel anlamda, bir hastalığı tedavi etmek ya da en azından bir hastanın klinik durumunu iyileştirmek amacıyla genetik materyalin hücrelere transferi olarak tanımlanır. Gen tedavisinin temel amacı, hedef hücrelere bir vektör aracılığı ile terapötik geni transfer etmektir. Gen tedavisinde en çok kullanılan vektörler, viral vektörlerdir. Adeno-assosiye virüs, retrovirüs, adenovirüs ve herpesvirüs vektörleri en sık uygulanan viral vektörlerdendir. Viral olmayan vektörler, viral vektörlerden daha az verimlidir, ancak düşük immünojenite ve büyük DNA parçalarını aktarabilmeleri onların avantajları olarak bilinir. Gen tedavisi hem somatik hem de eşey hücrelerinde uygulanılabilir. İlk kez 1982 yılında denenmeye başlayan, bir klinik uygulama olarak kabul edilen gen tedavisi, beklenenden daha hızlı gelişme göstererek çoğu bilim insanını kendine çekmiştir. Bu alandaki gelişmeler, ilk gen tedavisi ilaçlarının üretilmesini sağlamıştır. Örnek olarak kanser tedavisinde ve lipoprotein lipaz eksikliğinin tedavisinde kullanılması onaylanan ilaçlardan söz edilebilir. Ayrıca, Leberin konjenital amorozisi (LCA) ve hemofili hastaları için üretilmiş gen tedavisi ilaçları şu anda faz III aşamasındadır. Bu derlemede, gen tedavisinin temel ilkeleri, tarihçesi ve günümüzdeki klinik uygulamaları son literatür verileri ışığı altında sunulmaktadır.

The principles of gene therapy and recent advances

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.

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