American scientists have cured mice of a rare liver disease caused by a gene mutation. A new gene editing system based on bacterial proteins was applied.
The work, which is described in the journal Nature Biotechnology, proves that the method of genetic editing, known under the acronym CRISPR, can reverse the symptoms of disease in live animals. It assumes the extraction of mutated DNA and its subsequent replacement with the correct sequence. Scientists believe that technology has good potential for the treatment of many genetic diseases.
The most surprising thing is that we can actually correct damaged genes in live adult animals, leading lead researcher Daniel Anderson, a specialist in chemical engineering from the Massachusetts Institute of Technology (MIT), does not hide his joy.
The newly developed CRISPR system is based on the cellular defense mechanism that bacteria use against viral infections.
Scientists copied this system to create gene-editing complexes from the DNA-destroying enzyme Cas9 and the RNA guide strand, which is programmed to join a specific genome sequence to indicate to Cas9 the location of the "notch". When reconstructed, the cell copies the new genetic material from the template DNA chain into the genome.
There are other gene editing systems based on DNA-destroying enzymes (nucleases), but their use is associated with some difficulties, writes PhysOrg.
The CRISPR system is very easy to configure and configure. Other systems can potentially be used, but it’s much more difficult to get a suitable nuclease with them, ”explains a researcher by the name of Anderson, who also works at MIT.
Scientists hope that the technology they use once will help hemophilia, Huntington's disease, and other diseases caused by a unidirectional mutation.
The article is based on materials .
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