A Step towards Cystic fibrosis gene Therapy
Russian geneticists have successfully tested gene therapy for cystic fibrosis
Scientists from the Medical and Genetic Research Center in Moscow have developed and successfully tested the first Russian gene therapy for cystic fibrosis on cell cultures. Already now it surpasses foreign analogues, the press service of the FGBNU MGNC reports.
"Before proceeding to experiments with model animals and applying the technology on patients, it is necessary to conduct experiments on stem cells from other patients. I am sure that in a year we will talk about new results and successful development of therapy," said Svetlana Smirnikhina, head of the research group.
Cystic fibrosis is one of the most common diseases resulting from the appearance of single or multiple mutations in one particular gene. As a rule, it gives birth to about every three thousandth newborn, and its development leads to severe disorders in the work of the respiratory organs, intestines and many other organs.
The cause of its development is mutations in the CFTR gene, which is responsible for the production of sweat, digestive juices, mucus and other biological fluids in our body. At the moment, scientists know about two thousand different "typos" in this section of DNA that can cause the disease, which complicates the fight against it and the search for methods of its treatment.
Russian geneticists, as well as dozens of other scientific groups from foreign countries, have been working for many years to create a gene therapy capable of repairing at least some of the damaged copies of CFTR.
Recently, as scientists told at a press conference, they managed to make a big step in this direction. Using the CRISPR/Cas9 editor, geneticists replaced about one in ten damaged DNA sections in the culture of "reprogrammed" stem cells extracted from the body of a patient with cystic fibrosis.
In this regard, as the scientists noted, their methodology turned out to be noticeably more effective than its foreign "competitors". In other cases, geneticists managed to correct only every twentieth "typo" in the CFTR. The full results of all experiments with this version of gene therapy and its further prospects will be presented in peer-reviewed publications in the near future.
"Our article is currently being reviewed in one of the authoritative scientific journals. While we are not ready to say which one, but we hope that it will be published in the near future," Smirnikhina explained, answering questions from RIA Novosti.
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