Liver disease cured by DNA editing

  • Breaking
  • 23/04/2014

Scientists have managed to cure a disease in adult animals with a revolutionary genome-editing technique for the first time.

The technique allows scientists to make the smallest changes to the huge database of a DNA molecule.

Researchers have now had a breakthrough by using the technique to cure adult laboratory mice of an inherited liver disease but fixing a single 'letter' of the genome which had been mutated.

In adult humans a similar mutation in the same gene causes an equivalent inherited liver disease, and the treatment in mice increases hope that the same technique could be trailed in humans within a few years.

This is the latest breakthrough in genome editing, a branch of medicine that has been revolutionised since the discovery of Crispr technology, which allows scientists to alter almost any part of the genetic code precisely.

In the latest discovery, scientists at the Massachusetts Institute of Technology used the method to locate and correct the mutated gene pairin the liver gene known as LAH, which can be the cause of a fatal build up of amino acid tyrosine in humans, requiring drug treatment and a special diet.

The study's leader Professor Daniel Anderson said, "We basically showed you could use the Crispr system in an animal to cure a genetic disease, and the one we picked was s disease in the liver which is very similar to one found in humans."

source: newshub archive