How circular RNAs affect the muscleblind gene linked to muscle and nerve diseases
Official title: Unraveling regulatory functions of circRNAs at the muscleblind locus
Funded at Brandeis University · NIH-11252312 · Data updated 2026-11-12
This research looks at whether small circular RNA molecules control the muscleblind gene that plays a role in conditions like myotonic dystrophy and some forms of ALS.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brandeis University NIH-funded |
| Lab location | 1 site (Waltham, United States) |
| Project start | August 4, 2017 |
| Project end | December 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $327,198 |
| NIH project number | 5R01GM124406-07 |
| Project ID | NIH-11252312 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with myotonic dystrophy (especially DM1) or amyotrophic lateral sclerosis (ALS), or adults willing to provide muscle, blood, or other tissue samples for molecular studies would be most relevant.
✕Less likely to help
People without muscle or neurodegenerative conditions or children are unlikely to receive direct benefit from this laboratory-focused grant.
What this research studies
Researchers are studying tiny circular RNAs made from the muscleblind gene to see how they change gene activity in muscle and nerve cells. They use laboratory models such as fruit flies, cell systems, and tissue samples to track how circRNA production interacts with normal RNA splicing and protein levels. The team tests how altering circRNA levels changes muscle and neuronal function and related behaviors in those models. Understanding these molecular steps may point to new targets for therapies down the road.
Why it matters
Potential benefit: If successful, this work could identify new molecular targets to correct abnormal RNA processing in myotonic dystrophy and related neurodegenerative disorders.
How similar studies have gone: Prior laboratory studies, including work in fruit flies and cell models, have shown that circRNAs from the muscleblind locus can regulate gene expression and physiology, but translating these findings to human therapies has not yet been achieved.
Where this research is happening
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Brandeis University
Waltham, United States Active
Researchers
- Principal investigator: Kadener, Sebastian — Brandeis University
- Study coordinator: Kadener, Sebastian
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.