Scientists from the U.S. and Russia have created nanoparticles that are able to edit DNA

© Fotolia / angellodecoРабота laboratory for DNA researchScientists from the U.S. and Russia have created nanoparticles that are able to edit DNA© Fotolia / angellodeco

. Biologists from MIT, Skoltech, Moscow state University for the first time were able to connect promising genomic editor CRISPR/Cas9 nanoparticles, able to penetrate into cells of human and other living beings, and checked out their work on mice, according to a paper published in the journal Nature Biotechnology.

«I think the creation of fully synthetic nanoparticles that are able to turn on and off certain genes, will help us to not only get rid of genetic diseases of the liver and other health problems. Now we have a chance to overcome many diseases by correcting the genes in the bodies of adults,» said Daniel Anderson (Daniel Anderson) from the Massachusetts Institute of technology (USA).

In recent years, biologists have created several very accurate and promising editors DNA. They allow you to easily remove or replace individual genes, but leaves the rest of the genome, and even correct mutations in length in a single «letter»-nucleotide. This caused a revolution in the world of science, but breakthrough in the field of medicine, except for two very risky operations, has not happened yet.

The reason for this is that genomic editors need to deliver into the cell so that they can begin to «repair» DNA, by themselves, to get there they can’t. In vivo this problem is can be solved only one way — using a special retroviruses.

The retroviruses, there are many shortcomings that do not allow to apply them in medical practice. For example, space inside them is not enough in order to contain the most accurate version of the genome of the editors, and the immune system begins to recognize the virus after the first infection, so they cannot be used for repeat gene therapy.

Anderson and his colleagues, including Victor koteliansky and Timothy Zatsepin from Skoltech, Moscow state University, made the first step to solving this problem.

To create new nanoparticles, the researchers analyzed the structure of the various components of CRISPR, including «templates» for reading and cutting out genes, and found those parts, which can be attached to other molecules without disrupting their work. As it turned out, approximately 30% of letters in these templates and in the «blanks» protein Cas9 can be used to attach to the nanoparticles, without causing a significant reduction in their effectiveness.

Using these segments CRISPR/Cas9, scientists have prepared a set of fat nanoparticles are able to penetrate cells, and plated components of the genomic editor. These molecules are intended for removal of defective versions of the gene PCSK9 associated with abnormally high concentrations of blood cholesterol and premature death from diseases of the heart and blood vessels.

The work of these researchers tested the nanoparticles on mice. As shown by these experiments, the nanoparticles successfully penetrated and fixed the defect PCSK9 approximately 80% of the cells, which reduced the level of cholesterol in the blood of rodents by 35% and saved them from premature death.

Similarly «nanoverse» CRISPR/Cas9 can be used to combat other genetic diseases.