Therapeutic approaches for glutaric aciduria type I in adults
Official title: Therapeutic approaches for glutaric aciduria type I
Funded at Duke University · NIH-11600937 · Data updated 2026-11-12
Alicia and other adults with GA-1 could benefit from new liver-directed therapies and hepatocyte approaches to correct the faulty metabolism.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $593,848 |
| NIH project number | 1R56HD120276-01A1 |
| Project ID | NIH-11600937 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are adults (21+) with GA-1 or related metabolic symptoms who could participate in studies of liver-directed therapies or hepatocyte-based interventions.
✕Less likely to help
Patients without GA-1 or those in whom liver-directed therapy is not suitable may not benefit.
What this research studies
Glutaric aciduria type I is a metabolic disorder caused by a deficiency in an enzyme, leading to brain and organ problems. This grant focuses on therapies that fix the metabolic problem in the liver, which could prevent brain and kidney complications seen in GA-1. The researchers have shown in mice that restoring normal lysine breakdown in the liver can prevent harmful effects, and they plan to translate this to human treatment. The project aims to develop and test liver-directed gene therapy or healthy cell transplantation strategies to improve outcomes for adults living with GA-1.
Why it matters
Potential benefit: If successful, the work could lead to new treatments that reduce brain and organ damage and improve quality of life for adults with GA-1.
How similar studies have gone: Previous mouse studies suggest liver-directed approaches can correct the metabolic defect and improve outcomes, supporting potential human translation.
Where this research is happening
-
Duke University
Durham, United States Active
Researchers
- Principal investigator: Bissig, Karl-Dimiter — Duke University
- Study coordinator: Bissig, Karl-Dimiter
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.