American biologists have developed a specialized version of the Nobel Prize-winning gene-editing tool "CRISPR/Cas9", specifically designed to work inside the smooth muscle cells of blood vessels.
اضافة اعلان
Biologists in the United States presented the results of preclinical tests for an experimental gene therapy based on the modified version of the gene-editing tool CRISPR/Cas9, in an article published in the journal Nature Biomedical Engineering.
The researchers explained that they created a specialized version of the tool that significantly reduces the "typos" in DNA, which had represented a barrier to the safe use of the technology. Experiments on mice suffering from a human-like version of multisystem smooth muscle dysfunction syndrome — a deadly hereditary disease caused by a mutation in the ACTA2 gene — showed that the new therapy succeeded in correcting the mutation in 5–45% of cells, which prolonged the life of 60% of the rodents for more than six months, compared to the failure of all animals in the control group to survive beyond the first two months.
This rare disease leads to severe damage to the heart and blood vessels from an early age, including aneurysms, strokes, and failure in the aorta. Until now, there has been no effective treatment for it.
The researchers, under the supervision of Assistant Professor Kleinstiver at Harvard Medical School, confirm that these results represent a fundamental step toward developing a safe and effective gene therapy for such deadly genetic diseases.
