Doctors are hopeful that gene therapy could be a breakthrough for haemophilia, a blood disorder

On World Haemophilia Day, doctors highlighted the potential of gene therapy in treating haemophilia, a hereditary blood disorder. Haemophilia is caused by missing or defective clotting factors and raises the risk of severe bleeding and joint damage. Genetic counselling and screening are crucial for affected individuals. India has a high number of haemophilia cases, but many go undiagnosed due to lack of screening capabilities.

Gene therapy offers a promising approach to treating haemophilia by delivering functional genes to correct the genetic defect responsible for deficient clotting factor production. Recent clinical trials show positive outcomes, including the use of lentiviral vectors at CMC Vellore. While current treatment involves factor VIII infusions, gene therapy may offer a cure for haemophilia, especially in low and middle-income countries.

According to Anoop P, Sr. Consultant – Haematology at Aster RV Hospital, “Gene therapy is a potentially curative treatment for haemophilia.” It allows for editing the faulty gene of a baby inside the uterus, known to be born with haemophilia due to a family history. Ongoing research on gene therapy shows promising results, indicating a potential paradigm shift in the management and cure of haemophilia.