Gene disease fault corrected
Thursday October 13th, 2011
British scientists have made a fresh breakthrough in the search to find ways of correcting genetic diseases, it was announced last night.
Scientists say they have successfully corrected a faulty gene link to diseases of the liver and the lung.
The research has been conducted in the laboratory and not tested on patients yet.
But the aim is to generate thousands of cells that can be implanted in patients to combat genetic disease.
Scientists from Cambridge University and the Wellcome Trust Sanger Institute set out to correct a mutation in a gene called alphal-antitrypsin, which causes cirrhosis of the liver and emphysema of the lung. Currently the only treatment is a liver transplant.
Using a DNA splicing technique called piggyBac, they inserted a correct gene into the cells - and then proved that the right gene was in place.
Researcher Professor Allan Bradley said: These are early steps but, if this technology can be taken into treatment, it will offer great possible benefits for patients.
He explained: "We have developed new systems to target genes and integrated all the components to correct, efficiently, defects in patient cells.
"Our systems leave behind no trace of the genetic manipulation, save for the gene correction."
Fellow researcher Dr Ludovic Vallier said it was "a first step towards personalised cell therapy" for genetic liver disorders.
Targeted gene correction of ?1-antitrypsin deficiency in induced pluripotent stem cells. Yusa K, Rashid T et al. Nature, October 12 2011, doi: 10.1038/nature10424
Tags: Genetics | Internal Medicine | UK News
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