Scientists are about to deal with sickle cell anemia final. This will most likely achieved by editing the genome. At this stage, a research team from the University of Euna managed to achieve a remarkable result in elimination of the disease in mice using the method CRISPR. Although the result is not one hundred percent, for a first attempt it is highly encouraging. Sickle cell anemia arises from a mutation in the gene encoding the beta globulin, which is a component of hemoglobin. This leads to a change in the structure of the hemoglobin molecule and therefore the shape of erythrocytes, which has become sickle. This shape hinders the provision of erythrocytes in blood vessels, as a result of which can not satisfy the need of oxygen for the body. Normal erythrocytes resembles the shape of a double concave disk, which helps transfer and giving oxygen. Sickle cell anemia is a genetic disease that is transmitted in an autosomal recessive, meaning both parents possess and have passed the mutated gene. The disease is manifested by birth, as his establishment can be done immediately or later. Affected are characterized by fragile health.
In sickle cell anemia, the bone marrow produces pathological form of hemoglobin – S, instead of its normal A. erythrocytes have a relatively short life – he lives only 10-20 days instead of the usual 120. It also affects the life of the patients, except their health. One way of treating sickle cell anemia so far is the transplantation of bone marrow from a compatible healthy donor.
At this stage of the research team achieved genome editing using CRIPSR, in which was replaced in question mutated section of DNA. It has worked with donor blood from patients with sickle cell anemia. So far, scientists have not received modify all cells, but according to them even without 100% modification of red blood cells could be expected to substantially improve health in people suffering from this disease. The research of the scientists back into the bloodstream modified stem cells by this method in mice showed that red blood cells reach the optimal duration of life and circulate in the body four months. The study’s authors hope that by using the method in humans could be treated not only sickle cell anemia, but also other similar diseases. Forthcoming clinical trials involving volunteers over the next five years. According to the team is sufficient to achieve the replacement of even only 5% of damaged erythrocytes in the body in order to achieve a substantial improvement of health.