Disrupting Splicing Regulation to Rescue β-Catenin: A Novel Approach for Treating CTNNB1-Haploinsufficiency Disorder
Matea Maruna, Petra Sušjan, Maja Meško, Roman Jerala
This study uses spliceosome-mediated RNA trans-splicing to restore β-catenin production in CTNNB1 syndrome, showing improved trans-splicing efficiency with optimized PTMs.
bioRxiv · Apr 1, 2025 · PreprintMolecular Biology