GENE THERAPY AND GENE DELIVERY TO THE BRAIN USING VIRAL VECTORS
Hizli Sayar, Gokben
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Treating monogenic disorders via gene therapy although still considered experimental by some, has becoming a more accepted method lately especially in these last 10 years with a number of recent clinical successes. Genetic modifications are becoming easier to perform with the progressing technology and discovery of new techniques such as the Clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated protein 9 (Cas9) methods which can modify DNA with great ease and accuracy. Gene therapy is a powerful technique with huge potential to treat psychiatric and neurodegenerative disorders including Alzheimer’s and Parkinson’s disease. Gene therapy is simple in principle, which is corrective genetic material is sent into cells and the disease is cured by ending the problem at its source. Viral and non-viral vectors which are used for the delivery of the desired genes to the targeted cells are briefly listed and explained. Unlike viral vectors non-viral vectors don’t cause an immune response but their pretty low transfer rate makes them rather less interesting for research. Viral vectors of adenoviruses, adeno-associated viruses, retroviruses with its subclass of lentiviruses and herpes viruses are compared with their advantages and disadvantages related to usage in brain and CNS treatment of our topic. Neurotrophic factors (NTFs) have important roles in brain and nervous tissue. Delivering NTFs via viral vectors for treating neurodegenerative diseases is a promising approach. Providing information about principles, methods, hurdles and clinical applications of gene therapy with its historic background to present it with its all basic details and therapeutic effects it can provide to problems related to brain are aimed in this writing.