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First microRNA therapy for the heart works in humans

Copyright: Adobe Stock/ takasu

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A group of researchers from Goethe University and University Hospital Frankfurt demonstrates that an inhibitor against microRNA-92a is effective in humans. The German Centre for Cardiovascular Research (DZHK) supported this clinical trial.

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The microRNA therapy with antimiR-92a (MRG-110) proved a therapeutic effect in different experimental preclinical models. A current clinical study now indicates that the drug, which is also safe and well-tolerated in humans, inhibits microRNA-92a targets in the blood. This could improve heart function after heart attacks and other cardiovascular diseases.

MicroRNAs are short strands of RNA and can prevent or enhance protein formation. Since their discovery in 1993, scientists identified more than 1,900 microRNAs in humans that control a wide variety of biological processes and thus represent a promising therapeutic strategy in diseases. To date, microRNA inhibitors have already been successfully tested in initial clinical trials against hepatitis C and blood cancer.

In preliminary trials, anti-miR-92a has shown an increased formation of new blood vessels, improved blood circulation, and a positive effect on heart function. The substance also accelerates wound healing and protects against atherosclerosis, the narrowing, and inflammation of blood vessels.

The research group of Goethe University and University Hospital Frankfurt led by Prof. Stefanie Dimmeler, together with the Servier Institute and miRagen Therapeutics, has now been able to successfully test a potential microRNA therapy against cardiovascular diseases on humans for the first time. In healthy volunteers, the anti-miR-92a MRG-110 was able to inhibit microRNA-92a in the blood successfully. The results suggest that this inhibitor could become the first microRNA therapy for cardiovascular diseases in the future.

Original work: Efficiency and Target Derepression of Anti-miR-92a: Results of a First in Human Study; Nucleic Acid Therapeutics; Abplanalp et al., 2020; doi: 10,1089/nat.2020.0871.

Translational Research Project: Development of miR-92a inhibitors for the treatment of cardiovascular diseases