Scientists have cured liver disease mutated gene correction




American scientists have cured mice from a rare liver disease caused by a gene mutation. Was applied a new editing system based on bacterial gene proteins.

The work, which is described in the journal Nature Biotechnology, shows that the method of genetic editing, known under the acronym CRISPR, able to reverse the symptoms of the disease in live animals. Here it is assumed extraction mutated DNA and its subsequent replacement by the correct sequence. Scientists believe that the technology has good potential for the treatment of many genetic diseases.

The most amazing thing – is that we can actually adjust the damaged genes in living adult animals – does not hide his joy lead researcher Daniel Anderson, an expert in chemical engineering from the Massachusetts Institute of Technology (MIT).

At the heart of the newly developed cell CRISPR is a security mechanism that bacteria use against viral infections.

Scientists have copied this system to create genetically editing complexes of DNA-degrading enzyme Cas9 and chain guide RNA, which is programmed to join a particular genome sequence to indicate Cas9 place “cut.” When restoring cell gene copies in the new genetic material taken from the template strand of DNA.

There are other gene editing system based on DNA-destroying enzymes (nucleases), but their use is associated with some difficulties, writes PhysOrg.

CRISPR system is very easy to configure and customize. You can potentially use other systems, but they are much harder to get a suitable nuclease – explains the researcher by the name of Anderson, who also works at MIT.

Scientists hope that one day the technology used to help treat hemophilia, Huntington’s disease and other diseases caused by unidirectional mutation.
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Tags: MIT , Diseases , Genetic Engineering , Health , Medicine .

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