Novel gene therapy could reduce bleeding risk for hemophilia patients

Novel gene therapy could reduce bleeding risk for hemophilia patients uclnews nejm

that help prevent or stop bleeding. The gene responsible for making FIX protein is located on the X chromosome, so the severe form of hemophilia B is much more common in men.

Lead author Professor Pratima Chowdary says that"removing the need for hemophilia patients to regularly inject themselves with the missing protein is an important step in improving their quality of life. The long term follow up study will monitor the patients for durability of expression and surveillance for late effects."

While the treatment was generally well tolerated, all patients experienced some form of adverse events, with an abnormal blood clot in one who received the highest FLT180a dose and had the highest levels of FIX protein.

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