Experts from the Moscow Laboratory of Genome Editing at the Bochkov Medical Genetic Research Center have genetically engineered a mutation in the CFTR gene that leads to the development of cystic fibrosis. About it reports Medvestnik.
According to the publication, about 200 of the two thousand known to science ways of mutating the CFTR gene lead to the disease. Russian geneticists chose the F508del mutation, which is the most common in the European population.
To edit the genome, the CRISPR / Cas9 editor was used, which works on the principle of “molecular scissors” – to its creators this year handed over Nobel Prize. A short molecule delivers the Cas9 protein to the desired region of the genome, then Cas9 “cuts” the DNA of the mutated cells. If you add copies of the desired genes at the moment when the cell is trying to close the gap, then it will include them in its DNA.
The tests were carried out on the immortalized cell line CFTE29o- and pluripotent human stem cells with cystic fibrosis (cells of the patient’s skin, whose memory was “erased” and reprogrammed into stem cells). In the CFTE29o culture, the scientists managed to correct the mutation of about 3% of the cells, in the patient’s cell culture – 5%. In order to cure this disease, it is necessary to correct 10-15% of the cells, says Medvestnik. The next challenge for scientists is to improve this result.
Cystic fibrosis is a hereditary genetic disease that affects the human glands. Because of this, the processes in the body are disrupted, and the organs become susceptible to severe infections.
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