Exploring factors that influence TDP-43 in ALS and frontotemporal degeneration
Identification of TDP-43 modifiers through single-cell transcriptional and epigenomic dissection of ALS and FTLD-MND
This study is looking at a protein called TDP-43 that is connected to diseases like ALS and frontotemporal degeneration, to help us understand how it affects brain health and find new ways to help patients manage their conditions better.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project ID | NIH-10922705 on NIH RePORTER |
What this research studies
This research investigates the role of TDP-43, a protein linked to neurodegenerative diseases like ALS and frontotemporal degeneration (FTLD). By analyzing single-cell transcriptional and epigenomic data, the study aims to uncover the mechanisms behind TDP-43 mislocalization and its effects on neuronal health. Patients may benefit from insights that could lead to new therapeutic strategies to slow down or halt disease progression. The research employs advanced methodologies to dissect the genetic and pathological factors associated with these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with ALS or FTLD, particularly those with genetic mutations related to TDP-43.
Not a fit: Patients with neurodegenerative conditions unrelated to TDP-43 or those without a diagnosis of ALS or FTLD may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that slow or stop the progression of ALS and FTLD.
How similar studies have performed: Previous research has shown promising results in understanding TDP-43 pathology, but this specific approach is novel and aims to provide deeper insights.
Where this research is happening
Pittsburgh, United States
- University of Pittsburgh at Pittsburgh — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Donnelly, Christopher James — University of Pittsburgh at Pittsburgh
- Study coordinator: Donnelly, Christopher James
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.