Neurodevelopmental disorder–linked Argonaute mutations permit delayed RISC formation and unusual shortening of miRNAs by 3′→5′ trimming

Proceedings of the National Academy of Sciences, Volume 122, Number 46, November 2025.
Mutations in Argonaute (AGO) proteins cause Argonaute syndrome, a neurodevelopmental disorder (NDD), whose underlying molecular mechanism remains unclear. We determined the structures by cryo-electron microscopy (cryo-EM) of wild-type (WT) AGO1 and the…