About This Project
The brain has its own immune system. In C9 ALS/FTD, immune cells called microglia begin by protecting neurons, but at some point they switch and start causing harm. We believe this turning point represents a critical and therapeutically targetable moment in the disease. Our goal is to understand when and why this happens, and to intervene before irreversible damage occurs. By modulating inflammation and keeping these cells in their protective state, we aim not only to slow disease progression, but to fundamentally alter its course. Ultimately, we want to determine whether controlling this early immune shift can preserve neuronal function and meaningfully change outcomes for patients.
Lead Lab
Renzo Mancuso lab
Dr. Mancuso’s xenotransplantation model is the ideal in vivo system for assessing the contribution of C9 ALS/FTD to microglial dysfunction, and for modeling ways to remedy it using therapies that have the potential to change the disease course in humans.
What This Project Needs
- Funding to enable testing of immune-modulating therapies in C9 ALS/FTD models.
- Access to promising compounds from academia or industry to evaluate in our systems.
- Partnerships with immunology and drug development groups to advance candidate therapies.
- Our lab provides a platform to test drugs in human, disease-relevant models.
- If you work in neuroimmunology or drug development and have candidate compounds, this platform can move them forward.
- If you are a donor, this project offers a direct line from your contribution to drugs being tested in disease-relevant models.
Contact: Inquiries route directly to Renzo Mancuso’s lab.