STAUFEN 1 (STAU1) - MEDIATED MRNA DECAY

摘要

Disclosed are compositions and methods for treating subjects with conditions resulting from Stau 1 -mediated mRNA decay, screening for, and manufacturing those therapeutic agents. Described herein are novel mRNA decay mechanisms that involves mammalian Staufen (Stau)l, the nonsense-mediated mRNA decay (NMD) factor Upfl, and a termination codon. It is shown that Staul binds directly to Upfl and can elicit mRNA decay when tethered downstream of a termination codon. Also disclosed is the new pathway as a means for cells to down-regulate the expression of Staul-binding mRNAs.