Research Centre for Medical Genetics
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The Genome Editing Laboratory of the Research Centre for Medical Genetics became the first in Russia and the second in the world to obtain bronchial and pulmonary organelles

Specialists of the Genome Editing Laboratory of the Research Centre for Medical Genetics managed to obtain bronchial and pulmonary organelles from stem cells. Organelles are 3D structures made up of several types of lung cells. They can be used as models for scientific research, for example, to study the effects of various drugs. The Genome Editing Laboratory of the Research Centre for Medical Genetics is focused on working with the most common mutation in the CFTR gene in the European population, which leads to a severe hereditary disease, cystic fibrosis. The lungs and pancreas are primarily affected in this disease. In recent years, targeted drugs that can significantly change the quality of life of patients, have appeared. But these drugs are effective only for certain types of mutations. Editing the genome of somatic cells can change the approach to the treatment of all patients in the future, regardless of the type of mutation.

The technique for obtaining organoids from stem cells of patients with cystic fibrosis was optimized by Anna Demchenko, a researcher at the Genome Editing Laboratory of the Research Centre for Medical Genetics. Previously, she received a Presidential Fellowship for the research of technology for obtaining bronchial and pulmonary organelles and for editing the F508del mutation of the CFTR gene.

“In the next 10 years, we would like to be able to conduct the research in vivo: to transplant organelles obtained in our laboratory and observe their therapeutic effect. Confidence is inspired by successful experiments of our foreign colleagues that also work with bronchial and pulmonary organelles, on laboratory animals. Bronchial and pulmonary organelles have great perspectives in transplantation, for example. It is possible to seed transplanted lungs with organoids - as scientific studies show, this helps to increase the survival rate of the organ,” Anna said.

In addition to research on targeted drugs, organoids can be used to study the effectiveness of new genome editing techniques.

“The uniqueness of the technique for obtaining bronchial and pulmonary organelles lies in the fact that we use skin cells as a source,” explained Svetlana Smirnikhina, Head of the Genome Editing Laboratory of the Research Centre for Medical Genetics, Ph.D. “We use a small piece of skin from a patient with cystic fibrosis to obtain stem cells/ Then we apply certain cultivation conditions to these stem cells to obtain cells of the lungs and bronchi, organized into 3D structures - organelles. Thus, a lung model without using a lung biopsy is now available to us.”

The Genome Editing Laboratory of the Research Centre for Medical Genetics became the first in Russia and the second in the world to obtain bronchial and pulmonary organelles. The technology will receive a Russian patent.