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New research has found that a synthetic peptide (minihepcidins) may have a potential therapeutic effect on two serious blood diseases, beta thalassemia and polycythemia vera. Although the two kinds of diseases have the opposite effect on the generation of red blood cells, but minihepcidins can help restore normal levels of red blood cells, alleviate the abnormal increase in spleen; beta thalassemia, excessive accumulation of the drug can control iron ions, avoid serious toxic effects caused by excessive iron ion.
The author Stefano Rivella said: "a compound treatment and two distinct diseases seems to violate our intuition, but by limiting the absorption of iron, this compound can indeed in animal models in vivo red blood cell levels normalized. If these preclinical results can be translated into humans, it will provide a new treatment option for the treatment of these two blood diseases. "
In this study, the researchers investigated the therapeutic effects of minihepcidins on these two diseases using beta thalassemia and polycythemia vera mice models. In the case of beta thalassemia disease, genetic mutations damage the synthesis of hemoglobin, which affects the ability of red blood cells to carry oxygen, leading to anemia. However, due to low levels of hepcidin, the body continues to accumulate iron ions, which forms a vicious cycle - more and more red blood cells are damaged, and may lead to serious damage to the liver and heart.
In the case of polycythemia vera, a rare gene mutation drives the excessive production of red blood cells, forming a dense state of tomato sauce. This increases the risk of high blood pressure and thrombosis, and may cause strokes.
After careful study, the researchers found that in the young beta thalassemia mice model, minihepcidins can normalize the level of red blood cells and alleviate anemia and iron overload. In a slightly older mouse, the compound can improve the production of red blood cells and does not interfere with the effect of a chelating agent for the removal of excess iron deposition. In the model of polycythemia vera, minihepcidins can reduce the absorption of iron ions, reduce the number of red blood cells, but also alleviate the abnormal increase in spleen.
Minihepcidins is a modified hepcidin analog, which is able to simulate the role of hepcidin in the regulation of iron ions. The minihepcidins is smaller than the full length of hepcidin, but it has the characteristics of long term stability and lasting biological activity in animals. Previous studies have shown that minihepcidins treatment can prevent iron overload in a mouse model of hemoglobin deposition.