Scientists 'fix hemophilia in mice through genetic editing'

London , Mon, 27 Jun 2011 ANI

London, June 27 (ANI): Researchers using a new technique for editing the genome of living cells have shown that they can cure hemophilia in mice, a breakthrough which could help in new treatments for some hereditary diseases in humans.

 

They managed to persuade cells in mice to repair a faulty gene but, instead of recreating the flawed piece, the cells generated a healthy one.

 

The faulty gene was the one responsible for haemophilia, meaning that the process cured the mouse of the hereditary condition, which can be life-threatening in humans.

 

The scientists hope the discovery could help develop better treatments for conditions affecting the immune system, bone marrow and liver.

 

'Genetic editing' works by using enzymes to unlock the DNA.

 

A new gene could then be inserted in the right place and the DNA made to reform around it.

 

Dr Philippa Brice, of the PHG Foundation, a genetics think-tank based in Cambridge, described the research as "very promising".

 

The work was published in the journal Nature. (ANI)

 



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