THE ROLE OF GENETIC ENGINEERING AND GENE THERAPY IN THE TREATMENT OF HEREDITARY DISEASES

Abstract

This scientific work highlights the importance of genetic engineering and gene therapy in the treatment of hereditary diseases, their biological foundations, and prospects for application in modern medicine. Based on scientific sources, it was analyzed that due to achievements in genetics and biotechnology in recent years, the possibilities of diagnosing and treating hereditary diseases at the molecular level have significantly expanded. During the research, the role of genetic engineering in modifying genetic material, identifying defective genes, and replacing them with healthy genes was studied. Information was also presented about the effectiveness of gene therapy in treating hereditary diseases, especially in diseases such as hemophilia, cystic fibrosis, thalassemia, Duchenne muscular dystrophy, and spinal muscular atrophy. The study emphasized that CRISPR-Cas9 technology has created the possibility of highly accurate gene editing and initiated a new stage in the development of gene therapy. Along with the advantages of gene therapy, its biological and ethical problems were also analyzed. In particular, immune response reactions, incorrect gene integration, long-term safety issues, and high economic costs were identified as the main limitations of this technology. Nevertheless, it was scientifically substantiated that genetic engineering and gene therapy are among the most promising directions of future medicine. This scientific work noted that the rational use of genetic technologies in the treatment of hereditary diseases can significantly improve human health and reduce disability and early mortality. The research results are important for a deeper study of the role and prospects of gene therapy in modern medicine.