Promoter-targeted small RNA duplexes increase MBNL1 transcription and mitigate myotonic dystrophy-associated spliceopathy Abstract. Functional depletion of Muscleblind-like (MBNL) proteins is a key trigger of myotonic dystrophy (DM)-associated alternative splicing (AltS) defec

⭐NAR Breakthrough!⭐
🧬 New study shows that small activating RNAs can boost MBNL1 expression in #myotonicdystrophy cells, restoring splicing balance. This highlights #RNAactivation (RNAa) as a promising #therapeutic strategy for this #raredisease.
📖 doi.org/10.1093/nar/...
#NARBreakthrough