Using CRISPR technology to potentially cure a common kidney disease.
Official title: CRISPR-Cas Editing as a Genetic Cure for Autosomal Dominant Polycystic Kidney Disease
Funded at Nephrogen INC. · NIH-10822502 · Data updated 2026-11-12
This study is working on a new gene therapy for people with Autosomal Dominant Polycystic Kidney Disease (ADPKD) that aims to fix the gene causing the problem, which could help improve kidney health and offer a lasting solution for those affected by this condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Nephrogen INC. NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 19, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $325,000 |
| NIH project number | 1R41DK138689-01 |
| Project ID | NIH-10822502 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Autosomal Dominant Polycystic Kidney Disease, particularly those with mutations in the Pkd1 gene.
✕Less likely to help
Patients with other forms of kidney disease or those without genetic mutations related to ADPKD may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy for Autosomal Dominant Polycystic Kidney Disease (ADPKD), a genetic disorder that leads to kidney failure. The approach involves using CRISPR-Cas editing to correct mutations in the Pkd1 gene, which is responsible for most ADPKD cases. The researchers are optimizing adeno-associated virus (AAV) capsids to effectively deliver the gene therapy to kidney cells. If successful, this innovative treatment could provide a long-term solution for patients suffering from this debilitating condition.
Why it matters
Potential benefit: If successful, this research could offer a genetic cure for patients with Autosomal Dominant Polycystic Kidney Disease, potentially eliminating the need for dialysis or kidney transplantation.
How similar studies have gone: Previous research has shown promise in using gene therapy for genetic disorders, but this specific approach using CRISPR for ADPKD is novel and untested.
Where this research is happening
-
Nephrogen INC.
New York, United States Active
Researchers
- Principal investigator: Maxim, Demetrios S — Nephrogen INC.
- Study coordinator: Maxim, Demetrios S
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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.