Results in Hemophilia A Gene Therapy Trial Heralded as “Mind-Blowing”

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By Staff Writer

Factor VIII

A hemophilia study in the UK made headlines this week due to its “mind-blowing” results. BBC reports that the 13 person trial has given hope to these patients suffering from Hemophilia requiring daily factor VIII injections. They further state, “The virus is used like a postman to deliver the genetic instructions to the liver, which then starts producing factor VIII.”

From the article:

Prof John Pasi, who led the trials at Barts and Queen Mary University of London, said: “This is huge.

“It’s ground-breaking because the option to think about normalising levels in patients with severe haemophilia is absolutely mind-blowing.

“To offer people the potential of a normal life when they’ve had to inject themselves with factor VIII every other day to prevent bleeding is transformational.”

You can read the full article here.