Understanding ATP1A3-related illness using worm models to find new therapy targets
Official title: Identifying conserved cellular mechanisms involved in Alternating Hemiplegia of Childhood
Funded at Brown University · NIH-11373773 · Data updated 2026-11-12
Researchers are studying how faulty ATP1A3 genes cause Alternating Hemiplegia of Childhood and are using tiny worm models to find genes that could be targeted to treat the condition.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brown University NIH-funded |
| Lab location | 1 site (Providence, United States) |
| Project start | June 1, 2026 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $408,585 |
| NIH project number | 1R21NS142903-01A1 |
| Project ID | NIH-11373773 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Alternating Hemiplegia of Childhood or related ATP1A3-associated conditions that may benefit from future targeted therapies.
✕Less likely to help
People not affected by ATP1A3-related diseases may not directly benefit from this basic discovery work.
What this research studies
This project uses C. elegans models that carry human ATP1A3 mutations to explore how these changes disrupt cellular function. The team will screen genes that can suppress disease-related defects and examine cellular processes relevant to AHC. By identifying proteins and pathways linked to the condition, they aim to point toward potential therapy targets that could help people with AHC in the future.
Why it matters
Potential benefit: If successful, the work could uncover new targets for therapies that help manage or treat AHC.
How similar studies have gone: Previous studies in model organisms have shown that modifying certain genes can influence disease-related cellular problems, suggesting potential for this approach.
Where this research is happening
-
Brown University
Providence, United States Active
Researchers
- Principal investigator: Hart, Anne Church — Brown University
- Study coordinator: Hart, Anne Church
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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.