Using peptide nucleic acids to disrupt harmful RNA clusters in ALS and FTD
Official title: Peptide Nucleic Acid (PNA) Oligomers for Targeted Disruption of Biomolecular Condensates in ALS/FTD
Funded at Carnegie-Mellon University · NIH-10986735 · Data updated 2026-11-12
This study is looking at new ways to target and change certain RNA clusters in the brain that are linked to diseases like ALS and frontotemporal dementia, using special tools to help us understand how these clusters affect the disease and find new treatment options.
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 start | September 9, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $194,809 |
| NIH project number | 1R21NS141767-01 |
| Project ID | NIH-10986735 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with ALS or frontotemporal dementia who may benefit from novel therapeutic approaches.
✕Less likely to help
Patients with other neurological disorders unrelated to ALS or FTD may not receive any benefit from this research.
What this research studies
This research focuses on developing innovative molecular tools to target and manipulate biomolecular condensates (BMCs) that are implicated in diseases like ALS and frontotemporal dementia (FTD). By utilizing peptide nucleic acids (PNA), the researchers aim to fluorescently label specific RNAs involved in BMCs and prevent these RNAs from participating in harmful cellular processes. This approach allows for the study and potential disruption of these RNA clusters without altering the natural RNA sequences, which could lead to more accurate results. The goal is to better understand the role of these condensates in disease progression and explore new therapeutic avenues.
Why it matters
Potential benefit: If successful, this research could lead to new strategies for treating ALS and FTD by targeting the underlying molecular mechanisms of these diseases.
How similar studies have gone: While the approach of using peptide nucleic acids is innovative, similar strategies targeting biomolecular condensates have shown promise in other research areas, indicating potential for success.
Where this research is happening
-
Carnegie-Mellon University
Pittsburgh, United States Active
Researchers
- Principal investigator: Armitage, Bruce a. — Carnegie-Mellon University
- Study coordinator: Armitage, Bruce a.
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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.