Gene therapy for Aspartylglucosaminuria to improve neurological function
Official title: IND-enabling studies for Aspartylglucosaminuria (AGU) to support the initiation of an AAV9/AGA gene transfer clinical trial
Funded at Ut Southwestern Medical Center · NIH-10913596 · Data updated 2026-11-12
This study is testing a new gene therapy that aims to help people with Aspartylglucosaminuria (AGU) by delivering a healthy version of the faulty gene directly into the spinal canal, with the hope of improving brain function and slowing down the disease.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ut Southwestern Medical Center NIH-funded |
| Lab location | 1 site (Dallas, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $773,534 |
| NIH project number | 5U01NS129339-02 |
| Project ID | NIH-10913596 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old diagnosed with Aspartylglucosaminuria.
✕Less likely to help
Patients with other unrelated neurological disorders or those over the age of 11 may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy approach to treat Aspartylglucosaminuria (AGU), a severe genetic neurological disorder caused by a faulty AGA gene. The therapy aims to deliver a functional copy of the AGA gene using an AAV9 vector, which will be injected into the spinal canal. By restoring the enzyme's function, the research hopes to improve lysosomal function and reduce the harmful accumulation of substances that lead to neurological decline. This innovative approach is designed to address the underlying cause of AGU, potentially altering the disease's progression.
Why it matters
Potential benefit: If successful, this research could significantly improve the quality of life and neurological function for patients with Aspartylglucosaminuria.
How similar studies have gone: Previous gene therapy trials for similar genetic disorders have shown promising results, indicating the potential for success with this approach.
Where this research is happening
-
Ut Southwestern Medical Center
Dallas, United States Active
Researchers
- Principal investigator: Gray, Steven J — Ut Southwestern Medical Center
- Study coordinator: Gray, Steven J
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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.