Monday, April 16, 2012

UK - Haemophilia gene therapy breakthrough (UCL)

A gene therapy study developed by Dr Amit Nathwani (UCL Cancer Institute) and colleagues in collaboration with St. Jude Children's Research Hospital, Memphis, Tennessee, has shown that adults with haemophilia B benefit from the treatment, reducing need for injections with clotting factor to prevent bleeds. The findings of the six-person study mark the first evidence that gene therapy can reduce disabling, painful bleeding episodes in patients with the inherited blood disorder.

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