Abstract

THE FUTURE OF GENE THERAPY IN TREATING GENETIC DISEASES

Mohasin S.*, Jeffisha F., Sneha Susan Sunny and Sanoj Rajan

ABSTRACT

Millions of people worldwide are affected by rare genetic illnesses. The majority of them are brought on by genetic flaws that reduce quality of life and increase the risk of early death. Genetic therapies are seen to be the most promising kind of treatment for uncommon genetic illnesses since they work to correct or replace damaged genes.[8] Numerous treatments have recently received approval from international regulatory agencies.[6] Modern medicine's mainstay of the twenty-first century is gene therapy.[1] The creation of superior drug delivery vehicles is crucial to preventing the body from degrading gene therapy medications and successfully delivering them to target tissues, cells, and organelles.[17] Gene therapy has been proposed as a potential treatment for a wide range of illnesses, including cystic fibrosis, cancer, heart disease, hemopilia, diabetes, and AIDS.[6] Genetic illness treatment has never been easy. Only a small percentage of these illnesses are curable with specialised dietary regimens, chaperone molecules, or enzyme replacement therapy. Gene therapy has now been included to the list of treatments thanks to some recent
developments.[15]

Keywords: Gene therapy, vector, genetic diseases, AVV, Research.