Regenerative ideas for nerve health in Giant Axonal Neuropathy (GAN) through retinoic acid signaling
Official title: Regenerative Strategies Targeting Retinoic Acid Signaling in Giant Axonal Neuropathy
Funded at Univ of North Carolina Chapel Hill · NIH-11330543 · Data updated 2026-11-12
A patient-facing look at a research project that aims to improve nerve health in GAN by boosting a vitamin A-related signaling pathway.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of North Carolina Chapel Hill NIH-funded |
| Lab location | 1 site (Chapel Hill, United States) |
| Project start | May 14, 2025 |
| Project end | April 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $479,397 |
| NIH project number | 5R01NS136621-02 |
| Project ID | NIH-11330543 on NIH RePORTER |
Who could benefit from this research
✓Could help
GAN patients or individuals with similar genetic nerve conditions who could provide patient-derived cells or participate in future translational studies.
✕Less likely to help
People without GAN or related neurodegenerative conditions are unlikely to benefit.
What this research studies
This project uses GAN patient-derived nerve cells and a model mouse to study how retinoic acid signaling affects nerve degeneration. By focusing on specific RA-related proteins (CRABP1 and RARβ), researchers hope to uncover why GAN nerves deteriorate and how to restore healthy signaling. The work would help translate findings toward therapies that protect or repair neurons in GAN patients. The study combines human cell models with animal models to test potential regenerative strategies.
Why it matters
Potential benefit: If successful, the work could lead to new approaches that slow or reverse nerve degeneration in GAN.
How similar studies have gone: Past efforts using patient-derived neurons and RA signaling have shown promise in neural regeneration, supporting the approach's potential.
Where this research is happening
-
Univ of North Carolina Chapel Hill
Chapel Hill, United States Active
Researchers
- Principal investigator: Snider, Natasha T — Univ of North Carolina Chapel Hill
- Study coordinator: Snider, Natasha T
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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.