The U1 snRNP protein U1C and Helix H of U1 snRNA are critical for small molecule splicing modulator function
Zhiling Kuang, Brian Kosmyna, Xueni Li, Zhichao Tang, Shasha Shi, Karoline A. Lambert, Wenzheng Zhang, Joseph A. Giovinazzo, Kerstin A. Effenberger, Jairo Sierra, Lanqing Ying, Scott J. Barraza, Wencheng Li, Jingxin Wang, Christopher R. Trotta, Rui Zhao
This study shows that U1C and Helix H of U1 snRNA are essential for branaplam's effect on weak 5 splice site recognition, while risdiplam requires additional factors for activity.
Nature Communications · Jul 16, 2026Peer-reviewedBiochemistry