Developing small molecules to target faulty RNA structures in diseases
Design of Precision Small Molecules Targeting RNA Repeating Transcripts to Manipulate and Study Disease Biology
This study is exploring new tiny molecules that can specifically target and fix faulty RNA structures linked to various diseases, aiming to offer better treatment options for people with neuromuscular diseases and certain types of dementia.
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-11090350 on NIH RePORTER |
What this research studies
This research focuses on creating small molecules that can selectively target and manipulate malfunctioning RNA structures associated with various diseases. Current treatments often rely on oligonucleotides that can only target specific RNA sequences, which limits their effectiveness for many conditions caused by complex RNA structures. By designing small molecules that recognize these structures, the research aims to provide a more universal treatment approach for multiple diseases, particularly those related to RNA repeat expansions. This innovative method could potentially improve therapeutic options for patients suffering from neuromuscular diseases and genetically defined dementia.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients with neuromuscular diseases or genetically defined dementia caused by RNA repeat expansions.
Not a fit: Patients with diseases not related to RNA malfunction or those without the specific RNA repeat expansions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments for over 30 incurable neuromuscular diseases and genetically defined dementia.
How similar studies have performed: Previous research has shown success in targeting RNA structures with small molecules, indicating a promising avenue for further exploration.
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.