Scientists from the United States cured of multiple sclerosis using gene therapy

© Illustration RIA Novosti . Alina Planinata the artist imagined how the virus disables the immune cells that attack the brain of the patientScientists from the United States cured of multiple sclerosis using gene therapy© Illustration RIA Novosti . Alina Polyanina

American molecular biologists for the first time were able to rid mice of multiple sclerosis using gene therapy that causes immune cells to attack the brain of the patient, and published their findings in the journal Molecular Therapy.

«We have received very promising results – we were able to show that we can stop the disease, restore immune system to its original state and eliminate all the symptoms of multiple sclerosis. Although we conducted experiments on rodents, our findings show that the same therapy can be used for human therapy,» said brad Hoffman (Brad Hoffman) from the University of Florida in Gainesville (USA).

Multiple sclerosis is a disease of the nervous system in which immune cells begin to attack sheath of nerve fibers, called myelin. Without myelin the nerves conduct the signal worse and begin to «close», which leads to different consequences from mild numbness in limbs to paralysis or blindness. According to who statistics, over 2.3 million people suffer from this disease, effective treatments not yet invented.

Today dozens of research teams worldwide are working to develop treatments for this disease, and last year Russian scientists from the Institute of Bioorganic chemistry Russian Academy of Sciences proposed the first potential «vaccine» from multiple sclerosis, teaching immune cells to attack the brain and kill its cells.

Despite these successes, remains not clear why the immune system begins to consider the brain as «the enemy.» Some scientists believe that this is due to the penetration of infections into the brain, while others believe that the causes of multiple sclerosis are associated with disorders of the body and even with the presence of certain microbes in the gut microflora.

Hoffman and his colleagues created the first gene therapy that halts the disease causes the immune system to stop attacking the nerve cells creating the virus is able to penetrate the main «conductors» of the immune system, and get them to give a «signal extraction» for other immune cells, attacking the membrane of the neurons.

Typically, gene therapy, cannot be used to treat diseases of the brain, as viruses and other «means of delivery» of DNA fragments and proteins can’t penetrate the barrier between the circulatory system and Central nervous system. Biologists from the University of Florida were able to solve this problem by «aiming» the virus on brain cells, and liver.

The fact that the liver has unique immune cells which secrete special molecules, «reprogramming software an» regulatory T cells and other components of the immune system and forcing them to consider some of the antigens are part of the body. Infecting these cells with retrovirus, the scientists were hoping to get them to produce protein fragments, forming the basis of the myelin sheath of neurons, and «educate» T-cells not to attack them.

The virus the researchers tested a group of mice, which they have provoked the development of multiple sclerosis, by entering the toxin in their brain. Without treatment, the condition of such rodents progressive and very quickly getting worse, and after a few weeks they completely lose their mobility of the tail and limbs.

The introduction of the virus in their liver, as shown by the experiment, allowed the mice to cope with the disease and get rid of all its symptoms forever. Best virus worked in those cases when scientists additionally suppressed their immune system at the time when they introduced the virus into the body of rodents.

Successful completion of these experiments, as scientists hope, opens the way for clinical trials of their gene therapy and brings humanity to the creation of the first «working» cure for multiple sclerosis.

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