Advances in Gene Therapy and Modern Biotechnology

Abstract

Gene therapy and modern biotechnology have transformed the development of treatments designed to modify genetic information or the biological properties of cells. Contemporary approaches include gene replacement, gene addition, gene silencing, genome editing, and ex vivo modification of patient-derived cells. Viral vectors, particularly adeno-associated virus and lentiviral systems, remain important delivery platforms, while non-viral delivery, CRISPR-based editing, base editing, and other molecular technologies are expanding the range of possible interventions. The field has progressed from experimental concepts to an expanding class of clinically validated therapies, with regulatory approvals now covering rare genetic, hematologic, neurologic, dermatologic, and oncologic conditions. This paper examines nine major areas of progress, including gene replacement, vector engineering, genome editing, RNA-based approaches, cell-based gene therapy, biotechnology platforms, clinical applications, manufacturing, and future challenges.