Novel approaches for correcting gene expression insufficiency

A new molecular technology capable of binding to mRNA and regulating gene expression may offer a new avenue for treating diseases caused by haploinsufficiency.

The new technology consists of small molecules programmed to attach to speci one mRNA molecules to directly control their translation int Nature Communications di Messenger RNA udy offer a promising new strategy for treating a variety of diseases stemming from gene and protein expression insufficiency, researchers say. Northwestern University Journal 10.1038 2023 Nature Communica Alfred L. George Jr. cl MD s="summaryFeed_h Alfred Newton Richards l, RNA Yang Cao Chicagoland George SYNGAP1 Faculty at NorthwesternGeorge span class="summary the Center for Pharmacogenomics ge George lGi"> Bryan Dickinson Ph.D. lass="summaryFe Chemistry ghtT the University of Chicago SYNGAP1 RNA lGi">George PTEN ightText__NxlGi">only one ABCA7 hLightText__NxlGi">SYNGAP1 SYNGAP1 George half PTEN hLightText__NxlGi">taRNAs