Gene Therapy Shows Long-Term Success in Hemophilia B Patients

Gene therapy shows long-term success in hemophilia B patients

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years of follow-up data.

Hemophilia B gene therapy demonstrates long-term success

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years of follow-up data. Hemophilia B is a rare genetic disorder caused by insufficient levels of a circulating protein called factor IX, which promotes blood clotting.

Hemophilia B Gene Therapy Sustains Efficacy Over a Decade Later

(MedPage Today) -- A single infusion of adeno-associated virus (AAV)-mediated gene therapy led to sustained clinical benefit and no late-onset safety concerns in patients with severe hemophilia B, a longitudinal study showed. At a median follow...

Haemophilia B gene therapy safe, effective for long-term: Study –...

Last Updated on June 13, 2025 by Team THIP New Delhi, June 13 (IANS) 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, […] The post Haemophilia B gene therapy safe, effective for long-term: Study appeared first on THIP Media.

Researcher Foresees DNA Editing Becoming Common Medical Practice for Genetic Disease

Newswise — A West Virginia University expert said the custom gene-editing treatment that saved the life of a Pennsylvania baby with a rare genetic disorder — the first successful treatment of its kind in the world — could become common practice within the next decade. Dr. Abdulrazak Alali, a clinical and biochemical geneticist in the WVU School of Medicine Department of Pediatrics, explained that “bespoke” gene-editing therapy is individualized …