[ad_1]
Rome, July 9 (AdnKronos Health) – The use of genetic editing to inactivate a protein called Pcsk9 effectively reduces cholesterol levels in Rhesus macaques, a monkey species. Researchers from the Perelman School of Medicine at the University of Pennsylvania conducted the first study demonstrating a clinically relevant reduction in gene expression in a large animal model, thanks to the "molecular scissors", a search weapon against a variety of diseases and conditions. The team published this week's work on "Nature Biotechnology", paving the way for a possible new approach for the treatment of patients with heart disease who do not tolerate Pcsk9 inhibitors, the drugs used to fight against cancer. # 39; hypercholesterolemia. "Very often, these patients are treated with repeated injections of an antibody," explained the first author Lili Wang. "But our study shows that with genetic publishing, they no longer need this type of treatment." The co-authors of the survey, scientists at Durham's biotech company Precision Biosciences, have come up with an enzyme called meganuclease to recognize and specifically inactivate the Pcsk9 gene. A viral vector (adeno-badociated virus) was then used to transport the enzyme in the liver of primates. In treated animals, levels of Pcsk9 decreased between 45 and 84% and levels of Ldl ("bad" cholesterol in the blood) by 30 to 60%. Clinically relevant and stable reductions. Molecular badysis of liver tissue obtained from a biopsy also showed that mutations induce changes from 40 to 65% of Pcsk9 genes. Future studies will focus on mitigating immune toxicity and the appearance of changes in other genetic targets. But in addition to patients with hypercholesterolemia, these data open up hopes against a broad spectrum of metabolic diseases of the liver caused by mutations of different genes.
Source link
