Recently, in a research report published in the internationally renowned magazine Angewandte Chemie International Edition, researchers from the Scripps Research Institute (TSRI) have developed a new method by designing a molecule that can recognize and make RNA molecules Target inactive molecules to change the function of RNA in living cells. The researchers designed a new molecule that inactivates RNA and can cause dystrophic myotonia. Researcher Matthew Disney said that this new method is to use small molecules to inactivate RNAs in cells, which provides a new way to develop a series of new therapies. We all know that gene expression can be controlled by inducing the degradation of messenger RNA. Of course, we can also recruit a series of compounds to split these molecules. When a series of compounds can induce RNA division in vitro, so far, scientists have been RNA division has not been successfully induced in living cells.
Researchers have developed small molecules that can use RNA as a target to trigger type I myotonic dystrophy, accompanied by the production of molecules that produce hydroxyl radicals. When the small molecule recognizes the target, hydroxyl radicals are released to split the disease-associated RNA, thereby alleviating the symptoms of the disease. The researchers emphasize that although the compound can produce highly effective active free radicals, the compound is only non-toxic at relatively effective doses. The researchers said that for the first time, we have identified preferential RNA structural elements or sequences that bind small molecules, and then we observed these elements of disease-related RNAs, and developed molecules that bind to them to increase affinity to specifically target these. The component acts.
Type I dystrophic myotonia will cause the loss of RNA, which is known as the "triple repeat", which will cause a series of abnormal protein splicing. The variable symptoms of the disease include a series of muscle problems, cataracts, heart defects and Hormone changes, etc. The application value of this new research includes new cancer therapies combined with other therapies. This research can help researchers develop RNA function chemical probes or develop tools to detect RNA structure, which provides great treatment for related diseases. Help and hope.
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