Gene therapy for treating cystinuria and its effects on kidney health.
Genome engineering therapeutics for cystinuria and its metabolic consequences.
This study is exploring new gene therapies for cystinuria, a condition that causes kidney stones, to see if they can help reduce stone formation and improve kidney health, using advanced techniques tested in mice and lab-grown kidney cells.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Veterans Health Administration NIH-funded |
| Lab location | 1 site (Nashville, United States) |
| Project ID | NIH-11098437 on NIH RePORTER |
What this research studies
This research focuses on developing gene therapies for cystinuria, a genetic kidney disorder that leads to kidney stones. The approach involves using advanced techniques like adeno-associated virus (AAV) and transposon technology to deliver therapeutic genes directly to kidney cells. The research will test these therapies in mouse models and human kidney organoids to evaluate their effectiveness in correcting the underlying genetic defect. Patients may benefit from innovative treatments that could reduce kidney stone formation and improve kidney function.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with cystinuria, particularly those who are 21 years or older.
Not a fit: Patients with cystinuria who are under 21 years old or those with other unrelated kidney conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to effective gene therapies that significantly reduce the incidence of kidney stones and improve kidney health for patients with cystinuria.
How similar studies have performed: Other research has shown promise in using AAV technology for gene delivery in various conditions, indicating a potential for success in this novel application for cystinuria.
Where this research is happening
Nashville, United States
- Veterans Health Administration — Nashville, United States (Active)
Researchers
- Principal investigator: Wilson, Matthew H — Veterans Health Administration
- Study coordinator: Wilson, Matthew H
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 below.