Based on information from the Russian Assistance Foundation (Rusfond) official site
A method for treating cystic fibrosis based on genome editing is being developed in Russia. On January 14, Svetlana Smirnikhina, head of the Medical Research Center for Medical Genetics, spoke on the subject.
According to Smirnikhina, her laboratory was able to correct the most common mutation causing cystic fibrosis by using genome editing. Preliminary results show an effectiveness in mutation correction above 10%—more than twice the global success rate. This is the first experiment of its kind in Russia.
“We are sure that in a year, we will be discussing the successful results of genome editing, that we are close to developing a treatment for cystic fibrosis,” said Svetlana Smirnikhina.
According to preliminary assessments from the Research Center for Medical Genetics, after additional studies to confirm the lack of side effects, this method may be put into clinical practice in the next ten years. Svetlana Smirnikhina reported to Rusfond that the method consists of multiple stages. A special reaction turns cells, initially taken from an organism, into pluripotent stem cells. Then, using genetic editing, the mutation in the CFTR gene that causes cystic fibrosis is corrected. Researchers turn the mutation-free cells into bronchial epithelial cells, which are invulnerable to cystic fibrosis. Researches are currently working on the second stage: learning how to correct the most common mutations that cause cystic fibrosis.