Creating RNA treatments for TDP-43 related diseases in ALS and FTD
Developing RNA therapeutics for TDP-43 proteinopathy in ALS/FTD
['FUNDING_FELLOWSHIP'] · UNIVERSITY OF PENNSYLVANIA · NIH-10902056
This study is exploring new RNA-based treatments to help prevent the harmful clumping of a protein called TDP-43, which is linked to ALS and frontotemporal dementia, with the hope of finding better ways to help patients manage these conditions.
Quick facts
| Phase | ['FUNDING_FELLOWSHIP'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PENNSYLVANIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-10902056 on ClinicalTrials.gov |
What this research studies
This research focuses on developing RNA-based therapies to address the aggregation of TDP-43 protein, which is implicated in both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The approach involves studying how RNA can influence the behavior of TDP-43, particularly in preventing its harmful aggregation. By examining the interactions between TDP-43 and RNA, the research aims to identify potential therapeutic strategies that could mitigate the neurodegenerative effects of these conditions. Patients may benefit from innovative treatments that target the underlying molecular mechanisms of their diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with ALS or FTD, particularly those exhibiting TDP-43 protein aggregation.
Not a fit: Patients with other forms of dementia or neurodegenerative diseases not related to TDP-43 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new RNA-based therapies that slow down or halt the progression of ALS and FTD.
How similar studies have performed: Previous research has shown promise in targeting protein aggregation in neurodegenerative diseases, suggesting that this approach may yield beneficial results.
Where this research is happening
PHILADELPHIA, UNITED STATES
- UNIVERSITY OF PENNSYLVANIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: COPLEY, KATIE ELIZABETH — UNIVERSITY OF PENNSYLVANIA
- Study coordinator: COPLEY, KATIE ELIZABETH
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.
Conditions: Alzheimer's disease and related dementia, Alzheimer's disease and related disorders, Alzheimer's disease or a related dementia, Alzheimer's disease or a related disorder, Alzheimer's disease or related dementia