Hemophilia: gene therapy in success



According to an early study in the UK and the US Just one inoculation could be enough to mean people with hemophilia B no longer need medication. In the opinion of doctors the gene therapy was potentially life-changing




Researchers at University College London and St Jude Children's Research Hospital in the US were looking for a more permanent solution. They took a virus which infects people without symptoms adeno associated virus eight. It was then modified to infect liver cells with the genetic material for factor IX. Other researchers have described it as a truly a landmark study.

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