Role of GATA6 in SMA pathology
Official title: Defining the role of GATA6 in SMA pathology
Funded at Medical College of Wisconsin · NIH-11426569 · Data updated 2026-11-12
Aims to find new targets to help existing SMA therapies work better for kids and adults with spinal muscular atrophy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | September 11, 2026 |
| Project end | July 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $588,303 |
| NIH project number | 1R01NS150224-01 |
| Project ID | NIH-11426569 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with spinal muscular atrophy or those contributing samples to related glial/pathology studies, including biomarker discovery efforts.
✕Less likely to help
Patients whose SMA pathology does not involve glial-mediated mechanisms or whose disease is unrelated to SMN pathways may see limited benefit.
What this research studies
This project focuses on how the support cells in the nervous system may influence SMA progression and how GATA6 fits into that process. Researchers plan to study glial cells and their signals to understand non-neuronal factors that worsen motor neuron loss in SMA. By identifying how GATA6 and related pathways contribute to disease, they hope to uncover new therapeutic targets that could augment current SMN-restoring treatments. The work will use biologic models to explore mechanisms and potential combos with antisense oligonucleotide therapies.
Why it matters
Potential benefit: If successful, the research could lead to new treatment targets that enhance the effectiveness of SMA therapies and potentially slow disease progression.
How similar studies have gone: Previous work suggests glial factors influence SMA pathology, but targeting GATA6 as a therapeutic node is a novel approach with initial supportive data but awaits replication.
Where this research is happening
-
Medical College of Wisconsin
Milwaukee, United States Active
Researchers
- Principal investigator: Ebert, Allison D — Medical College of Wisconsin
- Study coordinator: Ebert, Allison D
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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.