ArticleNature communications2026
The U1 snRNP protein U1C and Helix H of U1 snRNA are critical for small molecule splicing modulator function.
Article in Nature communications, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Depleting prion protein using splice-switching small molecules.bioRxiv : the preprint server for biology · 2026Article
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16 authors.
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Abstract
Risdiplam and branaplam represent two classes of small-molecule splicing modulators that promote U1 snRNP recognition of weak non-canonical GA/GU-containing 5' splice sites (ss). We demonstrate that branaplam enhances recognition of these 5' ss by reconstituted U1 snRNP in vitro, and that this effect depends on the ZnF domain of U1C and Helix H of U1 snRNA, but not U1A or U1-70K. We also demonstrate that branaplam enhances the weak 5' ss recognition through a dual act of strengthening the U1 snRNP-5' ss interaction and U1 snRNP-U1C interaction. In cells, depletion of U1C reduces or abolishes compound-induced exon inclusion for most cassette exons. Interestingly, a subset of cassette exons become responsive to compound only upon U1C knockdown, supporting a model in which U1C stabilizes specific conformations at the 5' ss-U1 snRNA interface in a context-dependent manner that can either facilitate or hinder compound binding. Surprisingly, risdiplam shows no effect on weak 5' ss recognition in vitro, suggesting additional cellular factors are required for its activity.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.