When Luke Pembroke was diagnosed with the bleeding disorder haemophilia at 11 months old, doctors offered his mother Debra a glimmer of hope - one day there might be a cure.
For the paper, published in the New England Journal of Medicine, experts from UCL, Royal Free Hospital and biotechnology company Freeline Therapeutics trialled and continue to evaluate a new type of adeno-associated virus (AAV) gene therapy candidate
The novel gene therapy normalized and stabilized factor IX levels in 9 of 10 patients with moderate to severe hemophilia B, removing the need for regular injections.