Monday, December 12, 2011

Haemophilia gene therapy shows early success

"Just one injection could be enough to mean people with haemophilia B no longer need medication, according to an early study in the UK and the US.
Six patients were given a virus that infects the body with the blueprints needed to produce blood-clotting proteins. Four of them could then stop taking their drugs.
Doctors said the gene therapy was "potentially life-changing".
Other researchers have described it as a "truly a landmark study."
People with haemophilia B have an error in their genetic code, which means they cannot produce a protein called factor IX, which is critical for blood-clotting.
Patients are currently treated with factor IX injections, sometimes multiple times per week, but the manufacturing process is expensive.
Researchers at University College London and St Jude Children's Research Hospital in the US were looking for a more permanent solution.
Virus modification
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. The gene should then persist in the liver cells, telling the cells to manufacture the protein.
Six people were injected with the modified virus at the Royal Free Hospital in London. Two were given a low dose, two a middle dose and two a high level.
Results published in the New England Journal of Medicine showed levels of factor IX could be increased.
Normally, patients will have factor IX levels less than 1% of those found in people without haemophilia.
After injection, levels of factor IX ranged from 2% to 12%. The first patient treated has maintained levels of 2% for more than 16 months. One of the patients receiving the highest dose maintained levels which fluctuated between 8% and 12% for 20 weeks.
Carl Walker, aged 26 and from Berkshire, showed the greatest improvement. 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. (...)"

in BBC

4 comments:

Michael Westside said...

Gene manipulation with retro-viruses is always going to be awesome.

December 12, 2011 at 10:53 AM
Anonymous said...

I cant wait for gentically altered humans, bring on the futute baby
-anon mouse

December 12, 2011 at 3:56 PM
geld-grube said...

Interesting post! Think you have to be very careful with this but I'm looking forward to it :)

December 13, 2011 at 12:01 PM
Anonymous said...

biology, the most interesting subject :>

December 18, 2011 at 8:09 PM

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