EXPLORING THE POTENTIAL OF GENE THERAPY FOR INHERITED GENETIC DISORDERS

Abstract

Gene therapy is a revolutionary new development in biomedical research that has the potential to change the way inherited genetic illnesses are treated. This method is a big change from traditional medicines that just treat symptoms. It works by directly addressing and changing faulty genes at the molecular level. This study looks into the mechanics, uses, and clinical effects of gene therapy, with an emphasis on how CRISPR-Cas9 gene editing technology and viral/non-viral delivery vectors can work together. The strategy includes a thorough look at treatment trials, gene repair rates, and immune response profiles for a range of genetic disorders, such as cystic fibrosis, sickle cell anaemia, haemophilia, and muscular dystrophy. The results show that CRISPR-based gene editing is very good at fixing genes and reversing disease symptoms. On the other hand, viral vectors are better at delivering genes, but they also have a moderate risk of activating the immune system. Non-viral vectors are safer, but they don't work as well for transfection. The results of the trials reveal that gene therapy works over the long term in a wide range of people. This is shown by the fact that gene expression stability and clinical biomarkers increase consistently across trials. These results show that gene therapy is clinically viable and has therapeutic potential, but they also point out important problems such vector immunogenicity, delivery precision, and regulatory limits. For more people to use gene editing tools, they need to keep getting better, be safer, and be governed by ethical rules. Gene therapy is at the vanguard of personalised medicine. It gives promise for lasting solutions for genetic disorders that were once thought to be impossible to treat, and it will change the way we think about genetic healthcare in the future.