Novel approaches for correcting gene expression insufficiency
Originally published by Olivia Dimmer, Northwestern University, on November 17, 2023 taRNAs built from an array of IRESs increase reporter gene translation. Credit: Nature Communications (2023). DOI: 10.1038/s41467-023-42252-z A new molecular technology capable of binding to mRNA and regulating gene expression may offer a new avenue for treating diseases caused by haploinsufficiency , or the absence of one functional gene copy, according to a study published in Nature Communications . Messenger RNA, or mRNA , contains instructions for DNA to produce proteins. Many diseases , including cancer and many genetic disorders, result from insufficient gene —and therefore protein—expression, but few strategies exist to correct that kind of dysregulation at a molecular level. The new technology, dubbed "translation-activating RNAs" ( taRNAs ), consists of small molecules programmed to attach to specific mRNA molecules to directly control their translation into proteins,