Developing RNA treatments to address protein issues in ALS and FTD
Developing RNA Oligonucleotides to Mitigate Abberant FUS Phase Transition in FTD/ALS
This study is looking for new treatments that can help protect brain cells from a harmful protein linked to frontotemporal dementia and ALS, with the hope of improving the health of people living with these conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Thomas Jefferson University NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11075837 on NIH RePORTER |
What this research studies
This research focuses on finding new RNA oligonucleotides that can help mitigate the harmful effects of a protein called FUS, which is involved in the neurodegenerative diseases frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The approach involves understanding how FUS behaves under stress in cells and how it can form toxic aggregates that lead to cell damage. By developing agents that can reverse these harmful processes, the research aims to restore normal cellular function and potentially improve outcomes for patients suffering from these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with ALS or FTD, particularly those experiencing early to moderate symptoms.
Not a fit: Patients with advanced stages of ALS or FTD, where significant neurodegeneration has already occurred, may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic options that slow down or prevent the progression of ALS and FTD.
How similar studies have performed: While the approach of targeting RNA-binding proteins in neurodegenerative diseases is emerging, this specific strategy is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
Philadelphia, United States
- Thomas Jefferson University — Philadelphia, United States (Active)
Researchers
- Principal investigator: Guo, Lin — Thomas Jefferson University
- Study coordinator: Guo, Lin
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.