Improved models for ALS and frontotemporal dementia
The translational potential of next-generation sporadic mouse and human models of ALS/FTD
Researchers are creating better mouse and human models that mimic a key protein problem in ALS and frontotemporal dementia to help people with these conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of North Carolina Chapel Hill NIH-funded |
| Lab location | 1 site (Chapel Hill, United States) |
| Project ID | NIH-11251286 on NIH RePORTER |
What this research studies
This project makes new animal and human cell models that reproduce a specific problem with the TDP‑43 protein seen in ALS and FTD. The team used gene editing to create mice with an acetylation‑mimicking change in TDP‑43 and will compare those findings with human‑derived models and samples. They will study how the change causes protein clumping, RNA changes, and brain/behavior problems. The goal is to produce tools that more closely match the human disease so future therapies can be developed and tested faster.
Who could benefit from this research
Good fit: People diagnosed with ALS or frontotemporal dementia, or those willing to donate blood or tissue samples, are the kinds of patients who could be involved in the human-side components of this work.
Not a fit: Patients with unrelated neurological conditions or those seeking immediate treatment are unlikely to receive direct clinical benefit from this model-development research.
Why it matters
Potential benefit: If successful, these models could speed development of better-targeted therapies and improve testing of potential drugs for ALS/FTD.
How similar studies have performed: Other TDP‑43 models exist, but using an acetylation‑mimic change (K145Q) combined with matched human models is a relatively new approach that has shown promising preclinical signals.
Where this research is happening
Chapel Hill, United States
- Univ of North Carolina Chapel Hill — Chapel Hill, United States (Active)
Researchers
- Principal investigator: Cohen, Todd Jonathan — Univ of North Carolina Chapel Hill
- Study coordinator: Cohen, Todd Jonathan
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.