Transformational therapy cures haemophilia B

Transformational therapy cures haemophilia B A "transformational" therapy has effectively cured people with the bleeding disorder haemophilia B, say British doctors.

The treatment corrects a genetic defect that leaves people's blood struggling to clot and stop bleeding.

Elliott Mason, who was part of the trial which tested the therapy, says his life now feels "completely normal". BBC News

See also:

• Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B (abstract) The New England Journal of Medicine
• Haemophiliacs’ faulty gene could be cured with single injection The Daily Telegraph
• Gene therapy trial markedly cuts bleeding risk in haemophilia B patients The Guardian