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Genetic 'editing' breakthrough could lead to new treatments for diseases
By Fiona Macrae
Last updated at 1:26 AM on 27th June 2011
Comments (-) Add to My Stories Share Scientists have ‘edited’ the genetic code of life in a breakthrough that could lead to new treatments for diseases.
The discovery could lead to new ways of easing the symptoms and the pain of hundreds of conditions including haemophilia, cystic fibrosis and some hereditary forms of blindness.
For the first time, scientists have successfully ‘word-processed’ the DNA of a sick animal, returning it to health.
Breakthrough: Scientists have found a way to edit the genetic code of life which could lead to new treatments for hundreds of conditions
The mice treated had haemophilia, but with up to a third of genetic diseases caused by a single faulty gene, many more illnesses could potentially be treated this way.
The researchers, from the Children’s Hospital of Philadelphia in the US, used a two-step process to correct a genetic fault that stops blood clotting in haemophiliacs.
First, they used an enzyme described as a ‘molecular word processor’ to home in on the genetic flaw and break open the DNA, allowing it to be edited.
They then inserted a healthy gene in exactly the right place, the journal Nature reports.
The treatment was successful, with the animals’ blood clotting at near normal levels.
What is more, it lasted for the eight months of the study and appeared to be free of side-effects.
The idea of gene therapy, or the replacement of defective genes with health ones is not new.
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But scientists have struggled to find ways of inserting the ‘good’ gene in the right place. Put in the wrong position, it could trigger the development of cancer.
Study leader Dr Katherine High said: ‘We need to perform further studies to translate this finding into safe, effective treatments for haemophilia and other single-gene diseases in humans but this is a promising strategy for gene therapy.’
She now plans to test the treatment on larger animals. However, it will be at least a decade before the editing technique is widely used to treat haemophiliacs.
The condition is much more common in men than in women and affects up to one in every 5,000 baby boys.
Symptoms range from easy bruising to prolonged bleeding which, if internal, can be life-threatening.
Haemorrhages in the brain are particularly difficult to treat and can be fatal.
Treatment involves injecting a factor that clots the blood several time a week but can have side-effects and is too expensive for use in many parts of the world.
Dr Philippa Brice, of the PHG Foundation, a genetics think-tank based in Cambridge, said that there is a real need for new treatments.
She added: One of the dangers of gene therapy is that accidental insertion of healthy genes in the wrong place can be harmful, even causing cancer.
‘This ingenious approach, harnessing one of the body’s own genetic mechanisms, may be able to overcome this problem, and these initial results in mice are very promising.’
Read more: http://www.dailymail.co.uk/sciencetech/article-2008391/Genetic-editing-breakthrough-lead-new-treatments-diseases.html#ixzz1QZfxpB53