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Sunday 23rd October 2016

Gene therapy for haemophilia shows promise

12th December 2011

A type of gene therapy to treat patients with haemophilia B could mean one injection would prevent them from having to take medication.


Researchers in the UK and the US gave six patients a virus which infected the body with the tools required to manufacture blood-clotting proteins.

Four of the six were able to stop taking medication. Doctors said the results of the trial could prove "life-changing" for patients.

Haemophilia B causes a fault in a person's genetic code which means they cannot produce a blood-clotting protein called factor IX.

The treatment for the condition is injections of factor IX, but it is expensive and time-consuming.

The researchers, from University College London and St Jude Children's Research Hospital, were investigating how to find a permanent fix for the condition.

Six participants were given a modified virus at the Royal Free Hospital in London. Two received a low dose, two a medium dose and two a high dose.

Haemophilia patients usually have factor IX levels at less than 1% of people who do not have the condition.

However, after they were injected with the virus the patients showed factor IX levels of between 2-12%. 

Carl Walker, aged 26 and from Berkshire, showed the most impressive results following the treatment.

He said: "I have not needed any of my normal treatment, either preventative or on-demand as a result of an injury. Previously, I used to infuse at home three times a week."

"I play football, run and take part in triathlons - and previously I might have had to infuse both before I took part and possibly after as well. Not having to do that has been absolutely brilliant." 

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