AAV gene therapy for Alport syndrome
Official title: AAV vector-mediated gene therapy for Alport syndrome
Funded at Oregon Health & Science University · NIH-11456260 · Data updated 2026-11-12
A gene therapy approach using AAV vectors is studied to help people with Alport syndrome by restoring the missing collagen in the kidneys.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Oregon Health & Science University NIH-funded |
| Lab location | 1 site (Portland, United States) |
| Project start | September 1, 2026 |
| Project end | May 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $799,752 |
| NIH project number | 1R01DK144804-01A1 |
| Project ID | NIH-11456260 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Alport syndrome who have confirmed COL4A-related genetic mutations and are candidates for gene therapy trials.
✕Less likely to help
Participants with non-COL4A related kidney disease or those unable to tolerate gene therapy may not benefit.
What this research studies
This project focuses on a genetic approach to Alport syndrome, a condition that damages kidneys, ears, and eyes. It uses adeno-associated virus (AAV) vectors to deliver a healthy COL4A gene to affected tissues, aiming to restore the basement membrane where it’s deficient. Researchers will test the therapy in relevant cellular or animal models and explore safety and dosing considerations to pave the way for human trials. The ultimate goal is to slow or halt kidney disease progression and improve quality of life for patients with Alport syndrome.
Why it matters
Potential benefit: If successful, this therapy could slow kidney damage and reduce related complications for people with Alport syndrome.
How similar studies have gone: Previous gene therapy efforts in similar monogenic kidney diseases have shown promising safety signals and biological activity, though this specific approach is novel for Alport syndrome.
Where this research is happening
-
Oregon Health & Science University
Portland, United States Active
Researchers
- Principal investigator: Nakai, Hiroyuki — Oregon Health & Science University
- Study coordinator: Nakai, Hiroyuki
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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.