Mapping the RNA 'switches' that control gene messages
Large-scale characterization of the function of RNA regulatory elements
This project will map which RNA regulatory sites change how genes are edited and used, aiming to reveal targets for therapies for people with genetic conditions such as Duchenne muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project ID | NIH-11145034 on NIH RePORTER |
What this research studies
Researchers will test thousands of candidate RNA regulatory elements to find which ones change splicing, RNA stability, or protein production. They will use high-throughput lab experiments and computer analysis on human-derived cells and genomic datasets to identify functional sites. The team will perturb RNA-binding proteins and measure downstream effects on RNA processing and translation. The goal is to turn large catalogs of possible sites into a practical shortlist of targets for RNA-directed therapies like antisense oligonucleotides.
Who could benefit from this research
Good fit: People with genetic muscle disorders (for example Duchenne muscular dystrophy) who are willing to donate samples or consider future RNA-targeting treatments would be the most relevant candidates.
Not a fit: Patients whose conditions are unrelated to genetic errors in RNA processing or who need immediate clinical care are unlikely to benefit directly from this lab-focused research.
Why it matters
Potential benefit: If successful, this work could reveal new RNA targets that medicines such as antisense therapies can fix to treat genetic diseases like Duchenne muscular dystrophy.
How similar studies have performed: Targeting RNA elements has proven effective in at least one clinical case (Spinraza for spinal muscular atrophy), but comprehensive, large-scale mapping of functional RNA sites is a newer and still experimental approach.
Where this research is happening
Houston, United States
- Baylor College of Medicine — Houston, United States (Active)
Researchers
- Principal investigator: Van Nostrand, Eric Lyman — Baylor College of Medicine
- Study coordinator: Van Nostrand, Eric Lyman
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.