Research Information System
NUCLEOSIDES NUCLEOTIDES & NUCLEIC ACIDS, 2025 (SCI-Expanded)
Gene therapy has revolutionized the concept of treating genetic disorders by addressing the root causes at the genetic level, becoming one of the most quickly evolving fields in medicine today, especially due to its long-term effects. Gene therapy for the treatment of diseases relies on strategies of gene suppression, overexpression, and editing using different tools such as CRISPR and RNA interference. The gene transfer methods are broadly classified into three categories: physical, chemical, and biological. The use of viral vectors, such as adenoviruses, retroviruses, and adeno-associated viruses, is prevalent in clinical settings due to their high efficiency. Safety remains as an issue, and risk mitigation strategies will continue to evolve from clinical data to minimize complications related to gene silencing and immunotoxicity. In this review, various aspects of gene therapy have been covered, such as in-vivo and ex-vivo gene therapy, gene transfer methods, safety issues, as well as the gene therapy products approved until 2022. This review lists 35 licensed gene therapy products, detailing their therapeutic uses, mechanism of action, and vectors employed. Each product illustrates the various applications and potentials of gene therapy against untreatable conditions. Continuous improvements in gene transfer methods, vector safety, and clinical applications will increase the impact of the technology and offer hope for effective treatment and possible cures for different genetic disorders.