Context:
Scientists in India have reported success with using gene therapy to treat severe haemophilia A, a rare hereditary condition resulting from a faulty gene which triggers severe, spontaneous, and potentially fatal bleeding episodes.
Key Highlights:
- Hemophilia A is a rare, inherited bleeding disorder that occurs when the body doesn’t produce enough factor VIII, a protein that helps blood clot.
- No bleeding incidences were detected after an average 14 months of follow-up evaluation post-gene therapy in a trial led by Alok Srivastava of the Centre for Stem Cell Research (CSCR), Christian Medical College at Vellore.
- A one-time therapy is gene therapy and aims to introduce a gene into the body where that would produce enough amount of the clotting factor so that it will not bleed.
- Despite being rare in India, it is home to the second-largest population of patients who had someday 0.4-1 lakh estimates.
