Understanding how spinal muscular atrophy affects motor neurons
Official title: Identifying Developmental and Degenerative Mechanisms of SMA Using Single Motor Neuron Nucleus RNA Sequencing
Funded at Johns Hopkins University · NIH-10947636 · Data updated 2026-11-12
This study is looking into how spinal muscular atrophy (SMA) affects the nerve cells that control movement, with the goal of finding new ways to help people with SMA by understanding what happens to these cells during important growth stages.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | August 9, 2024 |
| Project end | August 8, 2026 |
| Funding year | 2024 |
| Award (this year) | $90,284 |
| NIH project number | 1F32NS138246-01 |
| Project ID | NIH-10947636 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with spinal muscular atrophy, particularly those experiencing significant neurological deficits.
✕Less likely to help
Patients with other unrelated neurological disorders or those without a diagnosis of spinal muscular atrophy may not benefit from this research.
What this research studies
This research investigates the mechanisms behind spinal muscular atrophy (SMA), a genetic disorder that impacts motor neuron function. By using advanced techniques like single nucleus RNA sequencing, the study aims to uncover how the lack of a specific protein affects the development and degeneration of motor neurons. The research focuses on understanding the differences in motor neuron behavior in SMA patients, particularly during critical developmental stages. This knowledge could lead to new therapeutic strategies that do not rely solely on existing treatments.
Why it matters
Potential benefit: If successful, this research could lead to the development of new therapies that improve motor neuron function and survival in SMA patients.
How similar studies have gone: Previous research has shown promise in understanding motor neuron diseases through innovative approaches, suggesting potential for success in this novel investigation.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Molotsky, Elana — Johns Hopkins University
- Study coordinator: Molotsky, Elana
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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.