Haemophilia gene therapy breakthrough (UCL)
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Uploaded by UCLTV on Dec 9, 2011
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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UCL press release: http://bit.ly/v5xtmE
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Would hemophiliacs who develop inhibitor be able to benefit from this treatment?
OlivierVid73 1 month ago
@OlivierVid73 - As only the producer of the video, I'm sorry that I can't answer your question. But if you want to read more, click on the link under the video to the UCL press release; that has a link to the original article in the New England Journal of Medicine, where this research was published. Good luck! I hope you find the answer. Rob
UCLTV 1 month ago
What about Haemophilia A - Factor VIII deficiency, can this treatment be applied to it in the same way?
Starsgoalie 2 months ago
@Starsgoalie - At the moment, no. Scientists do hope to apply this same method of gene therapy in the very near future to Haemophilia A, as well as diabetes and liver cancer.
UCLTV 2 months ago