GUWAHATI: Indian scientists have achieved a breakthrough with gene therapy to treat severe haemophilia A, a rare genetic disorder causing severe and potentially fatal bleeding episodes.
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The study, conducted on five patients in Tamil Nadu, showed positive results, with no bleeding episodes reported during an average follow-up of 14 months. The findings were published this week in the New England Journal of Medicine (NEJM).
The trial, led by Alok Srivastava of the Centre for Stem Cell Research (CSCR) at Christian Medical College, Vellore, was supported by the Union Department of Biotechnology. The mentioned therapy offers a one-time solution, introducing a gene that enables the body to produce sufficient clotting factor to prevent haemorrhages.
The study further employed a safer, non-adenoviral approach, potentially expanding its application to children.
Haemophilia A results from the absence of Factor VIII, a protein crucial for blood clotting. Incidentally, India is also home to the world’s second-largest haemophilia patient pool of 40,000–100,000 individuals.
