EFFECTIVENESS OF TREATING GENETIC DISEASES BY GENE THERAPY

Abstract

This article analyzes the effectiveness of gene therapy in treating genetic diseases, its main molecular mechanisms, and modern technologies. Key therapeutic approaches such as gene addition, gene function restoration, gene expression silencing, and targeted genome editing are covered. The potential of modern gene delivery and gene editing technologies, such as adeno-associated virus (AAV), lentiviral vectors, CRISPR-Cas9, base editing, prime editing, and lipid nanoparticles, is also considered. The results of modern research on the clinical efficacy of gene therapy in spinal muscular atrophy, hemophilia, hereditary retinal diseases, sickle cell disease, and beta-thalassemia are analyzed. Studies show that gene therapy can improve motor function, reduce bleeding, improve vision, reduce vaso-occlusive attacks, and achieve transfusion independence in some monogenic diseases. However, issues such as immunological reactions, off-target effects, vector limitations, long-term safety, high treatment costs, and proper patient selection for therapy were also discussed. In general, gene therapy is a promising treatment method that targets the molecular causes of genetic diseases, and its clinical effectiveness depends on the type of disease, the time of initiation of treatment, the characteristics of the genetic mutation, and the technology used.