Correct Answer : Hemophilia A
* 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.