Exploring ways to enhance nerve regeneration in giant axonal neuropathy using retinoic acid signaling.
Official title: Regenerative Strategies Targeting Retinoic Acid Signaling in Giant Axonal Neuropathy
Funded at Univ of North Carolina Chapel Hill · NIH-11132263 · Data updated 2026-11-12
This study is looking at how nerve damage happens in kids with giant axonal neuropathy (GAN) and is exploring ways to help nerves heal better by boosting a specific signaling process, which could lead to new treatments for those affected by this condition.
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 | 2025 |
| Award (this year) | $479,397 |
| NIH project number | 1R01NS136621-01A1 |
| Project ID | NIH-11132263 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with giant axonal neuropathy, particularly children affected by this condition.
✕Less likely to help
Patients with other forms of neuropathy or those without a diagnosis of giant axonal neuropathy may not benefit from this research.
What this research studies
This research investigates the mechanisms behind nerve degeneration in giant axonal neuropathy (GAN), a pediatric neurodegenerative disease caused by mutations in the KLHL16 gene. The team has discovered that retinoic acid signaling is significantly impaired in neurons from GAN patients, which may hinder nerve regeneration. By utilizing patient-derived stem cells and innovative mouse models, the research aims to understand how enhancing retinoic acid signaling can promote nerve growth and repair. This approach could lead to new therapeutic strategies for improving outcomes in patients with GAN.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that enhance nerve regeneration in patients with giant axonal neuropathy.
How similar studies have gone: While the specific application of retinoic acid signaling in GAN is novel, similar approaches in other neurodegenerative conditions have shown promise in promoting nerve regeneration.
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.