Understanding how a specific gene mutation causes ALS using rat models
Study on PFN1 Pathobiology Using Rat Models
This study is looking at how a specific gene mutation related to ALS affects the disease by using specially modified rats to help us understand what happens in the body, which could lead to better treatments for people with ALS.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Florida International University NIH-funded |
| Lab location | 1 site (Miami, United States) |
| Project ID | NIH-10604337 on NIH RePORTER |
What this research studies
This research investigates the role of the PFN1 gene mutation in causing amyotrophic lateral sclerosis (ALS) by using rat models. The study aims to determine how this mutation affects gene function at both systemic and molecular levels, which is crucial for understanding the disease. By creating a gene knockin model, researchers will introduce the mutation into the rat genome to observe its effects in a way that closely mimics human ALS. This approach allows for a detailed examination of the disease's progression and characteristics, which cannot be replicated in simpler models.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with a genetic predisposition to ALS, particularly those with known PFN1 mutations.
Not a fit: Patients without genetic mutations associated with ALS or those with other forms of motor neuron diseases may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a better understanding of ALS and potentially inform the development of targeted therapies.
How similar studies have performed: Previous research has shown success in using animal models to study ALS, indicating that this approach is promising and builds on established methodologies.
Where this research is happening
Miami, United States
- Florida International University — Miami, United States (Active)
Researchers
- Principal investigator: Xia, Xugang — Florida International University
- Study coordinator: Xia, Xugang
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.