Current Affairs - National - Discussion

India’s first human gene therapy, developed by CSCR at CMC Vellore, targets Hemophilia A, a genetic disorder causing blood clotting issues. The therapy, using lentiviral vectors, successfully reduced the annual bleeding rates to zero for five participants, eliminating the need for repeated injections. The results were published in the New England Journal of Medicine (NEJM) and show both long-term safety and efficacy. This breakthrough offers significant hope for the 136,000 patients suffering from Hemophilia A in India, marking a major step forward in the treatment of genetic disorders.