Nature sub-issue: Repairing the heart? Small molecule drugs that target RNA offer hope.

The good news is that, according to a recent study published in Nature Chemistry, a team led by Professor Matthew Disney, a chemist at the Scripps Research Institute in the United States, has found a small molecular compound that targets important biological molecules that cause a surge in expression levels in the failing heart muscle, providing a dawn for drug therapy for damaged heart tissue.
previous studies have found that vascular endotro cord growth factor A (VEGFA) can act as a signal to stem cells to rebuild blood vessels and muscles in damaged heart tissue and improve blood flow.
increased VEGFA expression may be a treatment for heart failure.
in this study, the team found that a microRNA pregenesome called pre-miR-377 increased expression levels in the failed heart muscle and inhibited the production of VEGFA.
, if this microRNA presuperve can be suppressed, it is possible to restart the VEGFA "production line".
because RNA molecules are small and not stable enough, RNA has long been considered an ideal target for small molecule drugs.
, despite this, Professor Disney et al. have developed a range of computing/chemical tools to overcome these obstacles, so they have targeted pre-miR-377.
using these tools, combined with a variety of compounds collected by AstraZeneca, the team began looking for "chemical partners" that could selectively combine the key conservative structural features of pre-miR-377.
not responsible for kung fu, they eventually found a small molecular compound called TGP-377.
Small molecule drug targets microRNAs and then raises VEGF schematics (Photo Source: Reference: Reference) So far, tests on TGP-377 in human vascular endotrative cell experiments have shown that this small molecule compound can target pre-miR-377, specific to raise VEGFA expression levels, created hope for the treatment of damaged heart tissue.
However, Professor Disney points out that since it has not yet been tested in animal models of heart failure, much remains to be done to turn TGP-377 into a potential drug that benefits a wide range of patients.
References . . . Heart repair factor boosted by RNA-targeting compound. Retrieved August 24, 2020, from .2. Hafeez S. Haniff, et.al, (2020). Design of a small molecule that molecules vascular endothelial growth factor A enabled screening by RNA fold-small molecule interactions. Nature Chemistry, DOI: 10.1038/s41557-020-0514-4.