How cells edit RNA messages (splicing)
Structure, regulation, and evolution of the splicing machinery
This research looks at how cells cut and join RNA messages so scientists can find new ways to treat cancers and genetic conditions like Duchenne muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Santa Cruz NIH-funded |
| Lab location | 1 site (Santa Cruz, United States) |
| Project ID | NIH-11323132 on NIH RePORTER |
What this research studies
From a patient's perspective, researchers are studying the molecular machinery that edits RNA before it becomes protein to understand why mistakes happen. They use engineered reporter molecules, biochemical tests, yeast cells, and RNA sequencing to observe splicing choices and a newly found quality-control pathway called "NO-BP decay." The team will perform gene-based suppressor screens, experiments in splicing extracts, and build predictive models of splicing capacity and competition between pre-mRNAs. These lab discoveries aim to reveal targets and rules that could guide future therapies to fix harmful splicing.
Who could benefit from this research
Good fit: People with cancers linked to splicing gene mutations or with Duchenne muscular dystrophy would be the most likely candidates for future treatments inspired by this work.
Not a fit: Patients whose conditions are unrelated to RNA splicing or caused by completely different mechanisms may not benefit from these findings.
Why it matters
Potential benefit: If successful, this work could point to new ways to correct harmful RNA splicing, potentially leading to therapies for cancers and genetic diseases like Duchenne muscular dystrophy.
How similar studies have performed: Some therapies that change splicing (for example, antisense drugs in Duchenne) have shown clinical success, but this project is basic research exploring new molecular mechanisms.
Where this research is happening
Santa Cruz, United States
- University of California Santa Cruz — Santa Cruz, United States (Active)
Researchers
- Principal investigator: Ares, Manuel — University of California Santa Cruz
- Study coordinator: Ares, Manuel
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.