Understanding and targeting the death of motor neurons in spinal muscular atrophy.
Mechanisms and Therapeutic Targeting of Motor Neuron Death in SMA
This study is looking at how motor neurons die in spinal muscular atrophy (SMA) to find new ways to help treat this condition, especially for babies and young children, by testing potential new drugs alongside existing treatments that boost a helpful protein.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-10771310 on NIH RePORTER |
What this research studies
This research investigates the mechanisms that lead to the death of motor neurons in spinal muscular atrophy (SMA), a condition that causes severe muscle weakness and is a leading genetic cause of infant mortality. The study aims to identify new therapeutic targets that can work alongside existing treatments that increase SMN protein levels, which have limitations in effectiveness. By using mouse models, the researchers will explore how motor neuron degeneration occurs and test potential drugs that could prevent this process. The goal is to enhance the overall treatment strategy for SMA patients by combining new therapies with current SMN-inducing approaches.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with spinal muscular atrophy, particularly those who may not respond adequately to current SMN-targeted therapies.
Not a fit: Patients with forms of motor neuron disease other than spinal muscular atrophy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that significantly improve outcomes for patients with spinal muscular atrophy.
How similar studies have performed: Previous research has shown promise in targeting neurodegenerative pathways in other conditions, suggesting that this approach could be beneficial for spinal muscular atrophy as well.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Pellizzoni, Livio — Columbia University Health Sciences
- Study coordinator: Pellizzoni, Livio
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.