Novel Therapeutics to Drive Gene Expression
When our genes do their job well, they keep us healthy. When our genes go rogue, however, they can turn on deleterious disease pathways and turn off our ability to fight disease. While controlling genes has long been a desirable goal for therapeutics, there is a new type of gene therapy that is gaining momentum: microRNA.
What are MicroRNA (miRNAs) and How Do They Work?
MicroRNAs are small, noncoding RNA molecules that can influence gene expression. How do they do that? Normally, a gene gives its orders to a messenger RNA. That messenger RNA then transcribes proteins that influence specific (good or bad) pathways.
Image Credit: By Kelvinsong https://commons.wikimedia.org/w/index.php?curid=23311105
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Here's the cool part. MicroRNA can bind to messenger RNA, effectively preventing messenger RNA from fulfilling a gene’s bad orders. And here is the even cooler part. Epitracker is discovering small molecules that appear to influence the expression of microRNA, which in turn, get genes back to doing their job well. Epitracker’s exciting line of small molecule therapeutic candidates that appear to influence microRNA could help to replace the hunt for traditional gene therapies for diseases currently without cures, such as muscular dystrophies.
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Epitracker's MicroRNA Library
To date, Epitracker has identified over a dozen small molecules in our compound libraries that independently predict changes in microRNA expression in vivo, coincident with improved health in diverse areas, including anti-aging, blood disorders, Alzheimer’s disease, and RNA-driven neuromuscular disorders. Our next steps are to assess the magnitude of desired changes in microRNA caused by our small molecules using human cell systems mimicking various disease states.