Which condition is targeted by India's first human gene therapy developed by CSCR at CMC Vellore?

A)
Hemophilia A
B)
Hemophilia B
C)
Thalassemia
D)
Sickle Cell Anemia

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.

Published On : December 12, 2024