Article
Translation of the intrinsically disordered protein α-synuclein is inhibited by a small molecule targeting its structured mRNA
3 Jan 2020
Abstract excerpt
Many proteins are refractory to targeting because they lack small-molecule binding pockets. An alternative to drugging these proteins directly is to target the messenger (m)RNA that encodes them, thereby reducing protein levels. We describe such an approach for the difficult-to-target protein α-synuclein encoded by the SNCA gene. Multiplication of the SNCA gene locus causes dominantly inherited Parkinson’s...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
