Developing treatments for brain diseases linked to TDP-43 protein changes
In vivo modelling and therapy development for stathmin-2 loss in TDP-43 proteinopathies
This research explores new ways to treat brain diseases like ALS, FTD, and Alzheimer's by focusing on a specific protein problem.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11121081 on NIH RePORTER |
What this research studies
Many neurological disorders, including ALS, frontotemporal dementia (FTD), and some Alzheimer's cases, are characterized by a protein called TDP-43 moving out of its normal place in brain cells and forming clumps. This disruption affects another crucial protein, stathmin-2, which is important for nerve growth and repair. When TDP-43 is disrupted, stathmin-2 levels drop, which is seen in most ALS/FTD patients. This project uses new mouse models to understand how the loss of stathmin-2 contributes to these diseases and to develop therapies that can restore its levels, aiming to help nerve cells regenerate and function better.
Who could benefit from this research
Good fit: This foundational research is for patients with Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), or Alzheimer's disease linked to TDP-43 protein changes.
Not a fit: Patients whose conditions are not related to TDP-43 proteinopathies or stathmin-2 loss may not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to new therapies that restore nerve cell function and slow or stop the progression of diseases like ALS, FTD, and Alzheimer's.
How similar studies have performed: Early studies in cell models have shown promise in restoring stathmin-2 levels, but this project explores these mechanisms and potential therapies in living models for the first time.
Where this research is happening
Boston, United States
- Massachusetts General Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Lagier-Tourenne, Clotilde — Massachusetts General Hospital
- Study coordinator: Lagier-Tourenne, Clotilde
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.