Using PNA molecules to target RNA clumps linked to ALS and FTD
Peptide Nucleic Acid (PNA) Oligomers for Targeted Disruption of Biomolecular Condensates in ALS/FTD
This project uses small synthetic molecules called PNAs to label and block disease-related RNAs that form clumps inside nerve cells in people with ALS or frontotemporal dementia.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Carnegie-Mellon University NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project ID | NIH-11187142 on NIH RePORTER |
What this research studies
Researchers will design PNA probes that bind specific RNA sequences tied to ALS/FTD and attach a fluorescent tag so the RNAs can be seen under a microscope. The team will test whether PNA binding prevents those RNAs from joining harmful biomolecular condensates by blocking RNA–protein or RNA–RNA interactions. Experiments will be done on cells and model systems using the natural (endogenous) RNAs so the targets do not need to be genetically altered. The probes are intended to be reversible so the researchers can add and remove them to study effects on condensate formation.
Who could benefit from this research
Good fit: People with ALS or frontotemporal dementia who are willing to donate samples or who may be considered for later clinical testing of PNA-based approaches would be the most relevant candidates.
Not a fit: Patients without ALS/FTD or those seeking immediate clinical treatments are unlikely to receive direct benefit from this laboratory-focused project.
Why it matters
Potential benefit: If successful, this could provide new tools to detect and block harmful RNA clumps, potentially guiding therapies that protect nerve cells in ALS and FTD.
How similar studies have performed: Antisense approaches have shown promise in ALS/FTD models, but applying PNAs specifically to visualize and disrupt RNA-driven condensates is a relatively new, preclinical idea.
Where this research is happening
Pittsburgh, United States
- Carnegie-Mellon University — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Armitage, Bruce a. — Carnegie-Mellon University
- Study coordinator: Armitage, Bruce a.
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.