Small molecules that destroy disease-causing RNA
Targeted degradation of RNAs by using small molecules
Developing small drug-like molecules that seek out and destroy disease-causing RNAs to help people with cancers such as Burkitt lymphoma, multiple myeloma, triple-negative breast cancer, and cervical cancer.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project ID | NIH-11301928 on NIH RePORTER |
What this research studies
Researchers are designing 'RiboTACs' — tiny molecules that bind a specific RNA and recruit the cell's own RNase L enzyme to cut that RNA. They have shown these compounds can lower levels of cancer-driving RNAs like MYC in lab-grown cancer cells and are testing them further in animal models and patient tumor cells. The team will optimize lead molecules for potency and selectivity and collect supporting data on effects and safety in preclinical systems. The aim is to turn this approach into drug-like options for targets that have been hard to treat with current therapies.
Who could benefit from this research
Good fit: People whose cancers are driven by specific RNAs such as MYC — including some patients with Burkitt lymphoma, multiple myeloma, triple-negative breast cancer, or cervical cancer — would be the most likely candidates for future clinical testing.
Not a fit: Patients without tumors driven by the targeted RNAs or those needing immediate standard-of-care treatment are unlikely to gain direct benefit from this laboratory-focused project right now.
Why it matters
Potential benefit: If successful, this approach could produce new drugs that directly remove cancer-driving RNAs and shrink tumors that are currently difficult to target.
How similar studies have performed: Early laboratory studies have shown RiboTACs can cleave oncogenic RNAs such as microRNA-21 and reduce MYC in cell models, but patient-level clinical success has not yet been demonstrated.
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.