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Hemophilia A
Context
Scientists in India have successfully used gene therapy to treat severe hemophilia A, a rare genetic disorder that causes spontaneous and potentially life-threatening bleeding due to a missing blood clotting factor.
How Gene Therapy Works?
- Traditionally, severe hemophilia A is treated by injecting a clotting factor called Factor VIII into the body regularly to prevent bleeding.
- However, gene therapy offers a one-time treatment. It involves introducing a new gene into the patient's body, which then teaches the body to produce enough Factor VIII on its own. This reduces the need for frequent injections.
- Comparison with Global Treatments: The only gene therapy approved for hemophilia A globally is Roctavian, which was approved by the U.S. FDA in 2023.
- It has shown to reduce bleeding incidents from 5.4 per year to 2.6 per year over a 3-year follow-up.
- However, Roctavian requires the use of corticosteroids to suppress the immune system for the therapy to work, and it uses an adenovirus to deliver the therapeutic gene.
- In contrast, the Indian trial used a different method. The gene was delivered using a lentivirus (a safer option than the adenovirus), and the therapy focused on fusing stem cells from the patients with the clotting factor gene.
- This approach is considered safer and might even be suitable for children, as it avoids the use of immunosuppressive drugs.
Hemophilia A in India:
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