"Gene scissors" are getting closer to people

CRISPR-Cas9 gene editing technology can become the basis for qualitatively new therapies, but the problem of its safety remains open. A new study on danio fish has shown that CRISPR-Cas9 does not produce gene mutations outside the target. These results confirm previous data obtained in animal models that when editing genes, the risk to the rest of the genome is minimal.

CRISPR-Cas9 is already being tested in early-stage clinical trials for the treatment of cancer, sickle cell anemia and blindness in children. To do this, researchers take cells from the body, edit the target gene in them and return them to the body.

Dr. Niko Katsanis and his colleagues from the Children's Research Institute named after Stanley Mann at Ann and Robert Lurie Children's Hospital in Chicago performed complete exome sequencing in more than 50 individual organisms from three generations of danio rerio fish and conducted thorough testing of the effects of gene editing. The danio fish is a widely used laboratory animal, 70% of its genome is similar to the human genome. Exome sequencing is used to identify genetic variants in genes that encode proteins that are the building blocks of cells, tissues and organs of the body.

This study is one of many in the field of gene editing safety. Its uniqueness lies in a large group of three generations of animals, which make it possible to track the non-targeted effects of gene editing not only in this individual, but also in descendants. In addition to clinically important results, the findings show that CRISPR-Cas9 is also a powerful research tool helping to create new models of genetic diseases.

Article by M.R.Mooney et al. Analysis of Single Nucleotide Variants in CRISPR-Cas9 Edited Zebrafish Exomes Shows No Evidence of Off-Target Inflation published in the journal Frontiers in Genetics.

Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru based on EurekAlert: CRISPR-Cas9 gene editing shows very low risk of mistakes.