Head of the Laboratory:
Svetlana A. Smirnikhina, MD, PhD
Alexander V. Lavrov, leading research scientist, MD, PhD
Olga A. Levchenko, research scientist, MD
Ekaterina V. Kondrateva, research scientist, MD
Milyausha I. Zainitdinova, research scientist
Arina A. Anuchina, research scientist, postgraduate student
Victoria A. Serzhanova, research scientist
Yana S. Slesarenko, research scientist, MD
Irina O. Panchuk, research scientist, MD
Anastasiya A. Poletkina, research scientist
Vladimir O. Sigin, junior research scientist
Daria A. Yurchenko, junior research scientist, MD
Elena V. Vasileva, laboratory assistant
Kirill D. Ustinov, laboratory assistant
Matvei I. Yasinovsky, laboratory assistant
The Laboratory of Genome Editing was organized on January 1, 2019 as a result of the separation of the genome editing group from the Laboratory of Mutagenesis.
The laboratory's efforts are focused on the topic of genome editing, including fundamental research for increasing the level of homologous recombination in CRISPR/Cas9 method, as well as applied research to develop treatment of hereditary monogenic diseases. The following works are carried out in the laboratory: 1) editing the most frequent mutation F508del in CFTR gene for the treatment of cystic fibrosis; 2) editing mutations in the DES gene for the treatment of hereditary cardiomyopathies; 3) single nucleotide editing of mutations in the KERA gene for the treatment of autosomal recessive Cornea plana disease.
Cystic fibrosis is one of the most common monogenic diseases. The electrolyte composition in the extracellular medium is disturbed due to mutations in the gene of the channel protein for chlorine ions (CFTR), so results to disruption of many organs. Pulmonary symptoms are dominant clinical features. Lungs’ pathology determines the prognosis and survival of the patients. Etiology-based therapy of this disease does not exist. Developed technologies of highly efficient target genome editing allow us to hope that such therapy will be worked out in the near future for many hereditary diseases. Using the technology of targeted nucleases CRISPR/Cas9, we are developing a method for correcting the most frequent mutation p.F508del in cystic fibrosis.