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.

This article was originally published on MedicalXpress.com

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