Cystic fibrosis in vitro
Russian scientists have obtained bronchial and pulmonary organoids for the first time
Press Center of the Ministry of Education and Science
Genome Editing Laboratory of the Medical and Genetic Research Center named after Academician N.P. Bochkova (MGNC) became the first in Russia and the second in the world to obtain bronchial and pulmonary organoids from stem cells.
Organoids are 3D structures consisting of several types of cells. They can serve as models for conducting scientific research, including for studying the effects of various drugs and testing genomic editing techniques.
The MGNC genome editing laboratory is focused on working with the most frequent mutation of the CFTR gene in the European population, which leads to the development of a severe hereditary disease cystic fibrosis, in which the lungs and pancreas are primarily affected.
In recent years, targeted drugs have appeared (they interfere with the mechanism of action of specific targeted molecules), which significantly change the quality of life of patients, but they 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 method of obtaining organoids from stem cells of patients with cystic fibrosis has been optimized by Anna Demchenko, a researcher at the MGNC Genome Editing Laboratory. A patent application for the technique has already been filed. Earlier, the scientist received a presidential scholarship to research the technology of producing bronchial and pulmonary organoids and editing the F508del mutation of the CFTR gene.
Various types of lung and bronchial cells stained with antibodies. Staining confirms that these cells are present in the resulting organoids.
"In the next 10 years, I would like to be able to conduct in vivo research: to transplant organoids obtained in our laboratory and observe their therapeutic effect. Bronchial and pulmonary organoids have great prospects, for example, during transplantation. It is possible to sow transplanted lungs with organoids — as scientific studies show, this helps to increase the survival rate of the organ," said Anna Demchenko. In addition to studies of targeted drugs, organoids can be used to study the effectiveness of new genomic editing techniques. "The uniqueness of the method of obtaining bronchial and pulmonary organoids is that their source is skin cells. We take a small fragment of skin from a patient with cystic fibrosis and get stem cells from it, which then, under certain cultivation conditions, become lung and bronchial cells organized into 3D structures — organoids. Thus, a lung model without lung biopsy is now available to us," concluded Svetlana Smirnikhina, Head of the MGNC Genome Editing Laboratory, Candidate of Medical Sciences.
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