Investigating new mouse and human models for ALS and frontotemporal dementia
The translational potential of next-generation sporadic mouse and human models of ALS/FTD
This study is creating special mouse models to better understand ALS and frontotemporal dementia, using a new technique called CRISPR, so that we can find better treatments for people living 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-11040651 on NIH RePORTER |
What this research studies
This research focuses on developing advanced animal models that better mimic the human conditions of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). By utilizing a novel approach involving CRISPR technology, the study aims to create mouse models that exhibit key features of these neurodegenerative diseases, including TDP-43 dysfunction. The goal is to enhance our understanding of the disease mechanisms and potentially identify new therapeutic targets. Patients may benefit from insights gained through these models that could lead to more effective treatments.
Who could benefit from this research
Good fit: Ideal candidates for participation or benefit from this research include individuals diagnosed with ALS or FTD, as well as their families.
Not a fit: Patients with other neurodegenerative disorders unrelated to ALS or FTD may not receive direct benefits from this research.
Why it matters
Potential benefit: If successful, this research could lead to breakthroughs in understanding and treating ALS and FTD, ultimately improving patient outcomes.
How similar studies have performed: Previous research has shown promise in using animal models to study neurodegenerative diseases, suggesting that this approach could yield valuable insights.
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.