Doctors have created a gene therapy that protects from atherosclerosis

© Photo : Illustration by RIA Novosti, Alina Polyaminopropyl editor cuts DNADoctors have created a gene therapy that protects from atherosclerosis© Photo : Illustration by RIA Novosti, Alina Polyanina

. American molecular biologists and physicians have developed a new type of gene therapy that dramatically reduces the level of cholesterol and other fats in the blood, damaging the ANGPTL3 gene, according to a paper published in the journal Circulation.

«Such a therapy would be particularly useful for people suffering from family hypercholesterolemia, a rare genetic disease, the development of which the level of cholesterol in the blood becomes too high. To treat her medically way is almost impossible. A single injection of CRISPR-«vaccines» that will be ready in the next five years, will help to get rid of it,» says Kieran Musunuru (Kiran Musunuru) from the University of Pennsylvania in Philadelphia (USA).

In recent years, scientists have made progress in the creation of various types of gene therapy that allows to remove individual genes and DNA segments that are associated with the development of various hereditary diseases, and replace them with the «fixed» version. For example, last year scientists have created a therapy to combat the degeneration of the retina, and two years ago they managed to stop the degeneration of muscles in the mice suffering from degeneration of muscles.

Musunuru and his colleagues used genetic engineering techniques to combat the main killer of people in developed countries and countries with economies in transition – heart disease and blood vessels, associated with the accumulation of plaque and other deposits on the walls of blood vessels.

According to current who statistics, approximately one in seven people in the United States and in other first world countries die from coronary heart disease, heart attacks and other diseases caused by atherosclerosis. Now, as scientists assume, the number of victims will grow rapidly in the coming years in connection with the General aging of the population and the spread of sedentary lifestyle and poor eating habits.

It tells Musunuru, large-scale genetic studies in recent years show that the Earth there is a relatively small population of people, which almost does not suffer from similar health problems because of mutations in one or two copies of the gene ANGPTL3.

This piece of DNA not only controls the growth of blood vessels and migration of cells, and also suppresses the activity of the two enzymes that break down the «bad» version of cholesterol and fat molecules, causing excessive activity leads to their accumulation in the bloodstream. Accordingly, mutations in ANGPTL3 lead to the opposite – the body breaks down almost all the stocks of fats in the blood, which protects their vehicles from atherosclerosis.

Following this idea, scientists have created a retrovirus on the basis of new genomic editor CRISPR/Cas9, which penetrates into the liver cells of mice or humans, ANGPTL3 and damage, and checked his work on the normal mice and their relatives, who suffered from hypercholesterolemia.

The first changes in the body of rodents appeared just two weeks after the introduction of the retrovirus – cholesterol and fats in their blood decreased by approximately 50-56%. These changes affected both healthy mice and their relatives with congenital disease, which allowed them to reduce the concentration of fats in their blood to relatively safe levels.

As noted by biologists, similar impressive results were obtained with rather modest efficiency of the virus – he managed to penetrate only one-third of the liver cells. Enhancing its «combat capability», according to Musunuru and his colleagues, will make such «vaccine» against atherosclerosis even more effective.

In the near future genetics plan to conduct the first tests of the «human» version of this gene therapy using mice whose liver is composed in part of cells of people. If these experiments succeed, then, according to Musunuru, you can think about the beginning of preclinical and clinical trials on volunteers.

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