What are the prospects of gene therapy and the latest methods of genome editing?

The biologist Dr. Doping tells about the methods of genome editing, CRISPR / Cas system and its application in medicine. Experts talk about promising research problems whose solution does not only produce scientific and technological breakthrough, but will also have a significant economic impact, such as the revolution in genetic engineering, gene therapy, and medical problems genome editing.

Editing of the genome, based on the CRISPR / Cas9 system - is a completely new technology that emerged only last year. The first publication of it appeared in early 2013, and during that time saw the light of hundreds of articles with examples of the use of this technique. In parallel, two other approaches are developed genome editing: ZFNs (Zinc-finger nucleases) and TALENs (Transcription activator-like effector nucleases). However, they are much more labor intensive, less elegant and more expensive.

Over the past 25 years, scientists have gradually discovered and explored completely new mechanism of immunity in bacteria, called CRISPR. The first work, which led ultimately to this outstanding discovery was published in 1987. However, the article at the time remained completely unnoticed. A significant breakthrough was the discovery of CRISPR when one of the variants of this system adapted to the cells of higher organisms.

The mechanism of CRISPR / Cas system is that the cut and double stranded DNA break is introduced into it. Moreover, the DNA molecule is cut in a very specific location, which is programmed that small RNA molecule, which is inserted into the cell. Today, examples of the use of this method, there are almost all types of organisms, and it is important that, in contrast to the traditional genetic engineering techniques which have been used for several decades and which arose after the opening-restriction enzymes enzymes capable, recognizing certain sequences in the DNA, do in it double-stranded break, CRISPR / cas9 allows you to make these genetic engineering in living cells.

In conventional cutting and genetically engineered DNA cross-linking held separately in vitro, in vitro, and after that the DNA introduced into the cell. This led in due time to the revolution, which ushered in biotechnology.

Breakthrough new feature consists in the fact that the same manipulations are carried out in a living cell, in which the cutting and insertion of the DNA molecule portion which is inserted from the outside into the cage. The genetic material of living cells is altered without the manipulation of the isolated DNA.

This opens up completely new perspectives and allows us to speak about the actual gene therapy, which is not futurological conjecture and reality. Along with the use of CRISPR to manipulate the genome of methods and ZFNs TALENS, which up to this point is mainly developed in the biotech industry, it was also brought to the specific applications. Who we developed an arsenal of techniques whereby animals (mice and even monkeys) have conducted a genetic disease therapy.

To improve immune system – buy Peptide complexes in solution Peptide complex 3, Prednisolon, Complex of cytamins for the immune system.

What is gene therapy? For example, you have a bad gene, and you want to replace with a good one. You enter this in a good gene DNA into the cell, and in the same CRISPR-introduced cell system that produces cutting DNA and short RNA molecule that directs the cutting mechanism, these "scissors" in the right place in the genome. These "scissors" cut out the bad gene, and in its place built a good. Such gene therapy is already being carried out on live animals, and they will recover.

The next stage is to bring these methods to use in medicine. Terms of these tasks depends on many circumstances. But, judging by some taken the start, examples of some specific applications in humans will be in a few years or even months. Primarily, these methods will be used for the treatment of fatal diseases that people have to die very soon. In the short term, gene therapy will depend on the severity of the disease. This may not be as common as plastic surgery. We intervene in the work of the cell, and there will always be side effects. The cell can turn into cancer.

CRISPR / Cas system is used not only for the treatment of genetic diseases. This gives a whole new impetus to the synthetic and systems biology, in an area where people modify the gene to cause the cell to do something new, to which she did not know how to do it.

Other medical uses CRISPR - the creation of animal models for medical purposes. It is the creation of animal models, which are endowed with properties similar to those of human patients. Using animal models we can study the disease and develop ways to treat it. With animals working is much easier than with people: removed a large number of prohibitions. Preparation of animal models with modified genome before the techniques have been developed with the use of CRISPR, it was very time-consuming, costly, demand the participation of highly qualified specialists. Now this process has become much easier, which is of great importance for modern medicine.