New Delhi, June 13 || A gene transfer approach to treating the bleeding disorder haemophilia B remains safe and effective long-term, according to a study by UK-based scientists.
Haemophilia B is a rare genetic disorder caused by insufficient levels of a circulating protein called factor IX, which promotes blood clotting.
Scientists from the St. Jude Children's Research Hospital and University College London used a one-time gene therapy intervention to address the disorder.
Their finding, based on 13 years of follow-up data, published in The New England Journal of Medicine shows an almost 10-fold reduction in annualised bleeding rate. It supports the long-term viability of gene therapy for the disease's treatment.
"The key benefit is that gene therapy is a one-time, simple intravenous infusion that's very straightforward to do and potentially has positive effects for a lifetime," said Andrew Davidoff, from the St. Jude Department of Surgery chair.
Haemophilia B is an X-linked genetic disorder affecting 1 in approximately 25,000 male births.