Understanding how spinal muscular atrophy affects motor neurons
Identifying Developmental and Degenerative Mechanisms of SMA Using Single Motor Neuron Nucleus RNA Sequencing
['FUNDING_FELLOWSHIP'] · JOHNS HOPKINS UNIVERSITY · NIH-10947636
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
| Phase | ['FUNDING_FELLOWSHIP'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JOHNS HOPKINS UNIVERSITY (nih funded) |
| Locations | 1 site (BALTIMORE, UNITED STATES) |
| Trial ID | NIH-10947636 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with spinal muscular atrophy, particularly those experiencing significant neurological deficits.
Not a fit: Patients with other unrelated neurological disorders or those without a diagnosis of spinal muscular atrophy may not benefit from this research.
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 performed: 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
BALTIMORE, UNITED STATES
- JOHNS HOPKINS UNIVERSITY — BALTIMORE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MOLOTSKY, ELANA — JOHNS HOPKINS UNIVERSITY
- Study coordinator: MOLOTSKY, ELANA
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.
Conditions: Aran-Duchenne disease