Improved CRISPR for the first time safely corrected DNA and cured fibrose16 октября, 2021
Now scientists see that the new technology can safely adjust DNA damage exclusively in the desired area. This is a big step forward in the future use of gene editing in clinical practice.
Mukobovysidosis is considered one of the most common genetic diseases in the world, therefore, in many countries, newborns are tested for mutation in the first days of life. Although there are ways of preventing the disease, the quality of life of such patients suffers. A group of researchers from the Netherlands studied CRISPR gene editing technology to solve the problem. Conclusions of their work
Scientists used PRIME editing — improved and safer CRISPR version. CRISPR / CAS9 works like genetic scissors, cutting through the CAS9 enzyme mutating the DNA fragment before correction, but this is associated with the risks of damage in other parts of the genome.
Experiments on human stem cells have shown that treatment fixes mutations and returns cells to a healthy state.
«We hope that in the future this will allow you to heal and prevent genetic diseases in humans. Our work is a big step towards clinical use of technology, «the author of Maarten Gersus concluded.
Meanwhile, scientists from the United States also
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