Viral gene therapies. AAV virus vectors in curing genetic diseases: overview, history and modern research

Abstract

Gene therapy has become an increasingly popular topic due to ongoing scientific research and clinical trials, which show promise in treating a wide range of diseases. This approach aims to cure genetic conditions previously deemed incurable by introducing new genetic material into targeted cells. Gene therapy now is considered also for many non– life-threatening conditions, including those adversely affecting a patient’s quality of life. [1] Viral vectors have been employed for the treatment of diseases such as metabolic, cardiovascular, muscular, hematologic, ophthalmologic, and infectious diseases and different types of cancer. [11] Gene therapy has been especially successful in the treatment of combined immunodeficiency syndromes, showing lasting and remarkable therapeutic benefits.