Precision small molecules that target repeated RNA in genetic neuromuscular and dementia disorders
Design of Precision Small Molecules Targeting RNA Repeating Transcripts to Manipulate and Study Disease Biology
This project creates small-molecule drugs that stick to harmful repeating RNA pieces that cause many neuromuscular and genetic dementia conditions, aiming to help people with those disorders.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project ID | NIH-11324512 on NIH RePORTER |
What this research studies
Researchers are designing small chemical compounds that bind the abnormally folded repeating RNA sequences that cause disease. The approach targets the RNA’s shape rather than its exact letter sequence, so one compound could help many different patients and diseases that share the same toxic repeat. The team has already seen these compounds work in lab and animal experiments to reverse disease features. This grant supports improving those molecules and moving them closer to treatments people could use.
Who could benefit from this research
Good fit: People with neuromuscular disorders or genetically defined dementias caused by expanded RNA repeat sequences would be the intended beneficiaries.
Not a fit: People whose conditions are caused by other kinds of mutations or non–repeat RNA problems are unlikely to benefit from this approach.
Why it matters
Potential benefit: Could lead to one type of drug that treats many different repeat-expansion diseases, providing new options for patients who currently lack effective therapies.
How similar studies have performed: The research team reports successful lab and animal results showing selective binding and reversal of disease-related effects, but human clinical benefits have not yet been reported.
Where this research is happening
Gainesville, United States
- University of Florida — Gainesville, United States (Active)
Researchers
- Principal investigator: Disney, Matthew D — University of Florida
- Study coordinator: Disney, Matthew D
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.