Gene editing technology crispr/cas9.

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The work performed by the student of Institute of Engineering Physics for Biomedicine (IEPB) NRNU MEPhI, group B16-301, Dept. № 35 "Medical physics", Tikhonovsky Gleb. Specially for PhysBio Summer School “Physics and technologies in life sciences”. July 2017.

GENE EDITING TECHNOLOGY CRISPR/Cas9.

CRISPR/Cas9 is a relatively new technology for editing genomes of higher organisms based on the immune system on the contrary the simplest organisms - bacteria. This technology can change our attitude to a great many widespread hereditary diseases. If earlier they were either incurable, today it is possible to directly resolve the very cause of the disease.

With the advent and development of technology for genome editing you will also be able and its "improvements", changes in their own interests. For example, the editing may not be the only genes associated with hereditary diseases, but also genes related to increased risk for health or responsible for success in sport, the ability to be receptive to the most common allergens: plant pollen, dust, animal hair, lactose...

I. GENERAL INFORMATION ON CRISPR/Cas9 TECHNOLOGY.

At the core of this system is the DNA of bacteria - CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). These sites are available in almost all archaea and most bacteria. They can be located directly on the chromosome, which happens often, and in the composition of phages (viruses of bacteria) that is found less frequently. This system (CRISPR – Cas) are composed of two main parts: the CRISPR-cassettes and the adjacent gene cluster Cas. Cassette is a block complementary sequences, repeats with a length of 24 to 48 nucleotides. These repeats contain a so-called spacers — a unique insertion of about the same length as the repetition. The spacers are identical to various parts of the phages, and other elements ever penetrated into this cell or cells of her ancestors. Thus, the CRISPR can be considered as a kind of archive, album, carefully keeping information separated by the repetition, about the pests and just any "visitors" ever violate the boundary of the cell and to oppose new intervention, the archive must reside in your state and constantly updated.

In the beginning of XXI century group of scientists were compared to sequences of known CRISPR spacers from DNA sequences. It turned out that quite often the sequence of spacers were similar to sequences of viruses. That in turn suggests that CRISPR-cassettes can carry a protective function. At the same time, was detected Cas genes are often located in close proximity to CRISPR-cassettes. And then, on the basis of the received data, scientists have developed a model in which, after contact with the virus in the cell he'd found it in the Cas protein using synthesized c CRISPR RNA copy. And if any of the fragments of the genome of the virus coincided with the recorded in the spacer, then Cas cuts the viral DNA and causes a chain reaction in which all the DNA would be destroyed.

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