Scientists have taken an important step in the treatment of genetic diseases: for the first time, they have used CRISPR-Cas9 technology to remove the extra copy of chromosome 21 in cells with Down syndrome.
This is one of the most common genetic conditions, affecting about one in 700 newborns. Its cause is trisomy 21, meaning that there are three copies of this chromosome instead of two. Until now, medicine has not been able to correct the root cause of the problem.
The research was carried out on cellular models from stem cells and skin cells. Scientists were able to ensure that one maternal and one paternal copy of chromosome 21 remained in the cell. As a result, the cells began functioning again as they do in a healthy body.
For now, the technology has only been tested in the laboratory, and it is still too early to talk about using it in the human body: there is a risk of random changes in other chromosomes. However, the study confirms a global trend: genome technologies are becoming more precise and are beginning to solve problems that until recently seemed unsolvable. If progress in CRISPR continues at this pace, the concept of an "incurable genetic disease" may gradually disappear.
