19 February 2020

Genetically modified leukoconcentrate

A drug for gene therapy from the patient's blood has been developed in Russia

RIA News

Scientists from Kazan State Medical University have developed and patented a new drug – a genetically modified leukoconcentrate. This remedy based on the patient's blood cells and human therapeutic genes will help fight degenerative, ischemic and infectious diseases. The research is carried out with the support of the Russian Science Foundation (RNF).

Nowadays, gene therapy is becoming increasingly popular. This method of treatment involves the introduction of "healthy" genetic material into the body that can restore or replace defects in the patient's DNA in order to eliminate the cause of the disease, not its symptoms. In case of successful treatment, restoration of the function of the damaged gene is observed.

However, gene therapy also has risks – possible malignant transformation of patients' cells. This fact is one of the main obstacles to the widespread introduction of gene therapy.

"Gene therapy approaches are being introduced into practical medicine rather slowly due to high risks and costs," Rustem Islamov, the head of the study, Professor of Kazan State Medical University, is quoted in the RNF press release as saying. – Correction of pathological processes with the help of genetically modified leukoconcentrate can become one of the breakthrough directions in gene therapy."

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Scheme of preparation and application of genetically modified leukoconcentrate

The method proposed by scientists solves another problem of gene therapy related to the delivery of genetic material. Today, there are two main ways to deliver therapeutic genes: with the help of harmless modified viruses that have natural mechanisms for introducing new information into another organism, or with the help of liposomes – fat bubbles. Gradually, a third one is entering medical practice, using white blood cells as a transporter for artificial DNA – white blood cells. In the body, these cells act as protectors against external and internal pathogens, and they are easily obtained from the patient's blood.

Kazan scientists have developed a simple, safe and inexpensive way to get a kind of individual drug for each patient – a genetically modified concentrate of white blood cells that carry new information in the form of artificial DNA.

The technology of this personalized drug is as follows. Leukocytes are isolated from the patient's whole blood using a special starch. Then a therapeutic gene or a combination of genes is added as part of a previously modified and therefore harmless virus. After that, the drug is injected back into the patient's blood. White blood cells easily mix with blood and penetrate into different tissues without causing an immune response. And the genetic material that they transport ensures the production of proteins necessary for the patient.

Now scientists are conducting test studies of the drug in spinal cord injury on mini-pigs.

In the future, the use of technology will allow people to cope with the consequences of a brain stroke, nerve cell injuries and diseases of the nervous system, strengthen immunity in viral diseases, correct blood clotting disorders, increase the rate of bone tissue regeneration and not only – depending on the therapeutic genes that will carry leukocytes.

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