Using a new system of genetic editing based on bacterial proteins by researchers from MIT cured rare liver disease caused by a single genetic mutation.
The findings described in the edition of Nature Biotechnology, provide the first evidence that the technique of editing of a gene known as CRISPR, can reverse disease symptoms.
CRISPR, which offers an easy way to crop the mutated DNA and replacement with the correct sequence has the potential to treat many genetic diseases, according to the research team.
Recently developed CRISPR system relies on cellular mechanism that bacteria use to protect themselves from viral infection.
Researchers have copied this cell system for the creation of gene-editing complexes, including DNA.
They are cut enzyme called Cas9, bound to the RNA strand, which can be programmed to bind to a specific genomic sequence.
Meanwhile, researchers deliver DNA template strand.
When repairing cell damage resulting from Cas9, scientists introduced the template DNA in the genome.
Scientists predict that this type of revision of the genome one day could help in the treatment of diseases such as hemophilia, Huntington's disease, and the like, caused by a single mutation.
There are other systems developed on the basis of genetic editing of DNA enzymes known as nucleases, but these complexes can be expensive and difficult to assemble.
In contrast, CRISPR is very easy to configure and customize equipment.