Developing personalized gene therapies for liver disorders caused by genetic mutations
Official title: Personalized prime editing as a platform for hepatic inborn errors of metabolism
Funded at Children's Hosp of Philadelphia · NIH-11223000 · Data updated 2026-11-12
This study is looking at a new way to create personalized treatments for rare liver disorders caused by genetic issues, like phenylketonuria (PKU), to help patients avoid the need for liver transplants.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 20, 2024 |
| Project end | August 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $1,427,560 |
| NIH project number | 3U01TR005355-01S1 |
| Project ID | NIH-11223000 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with severe hepatic inborn errors of metabolism, particularly those with phenylketonuria or similar conditions.
✕Less likely to help
Patients with non-hepatic metabolic disorders or those whose conditions do not involve genetic mutations affecting liver function may not benefit from this research.
What this research studies
This research focuses on creating personalized prime-editing therapies for rare liver disorders known as inborn errors of metabolism (IEMs). These conditions arise from genetic mutations that affect the liver's ability to process certain substances, leading to harmful accumulations in the body. The approach involves using adeno-associated viral vectors to deliver specific gene-editing tools tailored to individual patients' genetic variants. By targeting conditions like phenylketonuria (PKU), the research aims to provide a new treatment option that could potentially replace the need for liver transplants.
Why it matters
Potential benefit: If successful, this research could offer a groundbreaking treatment for patients with severe liver disorders, significantly improving their quality of life.
How similar studies have gone: Previous research has shown promise in using gene editing techniques for metabolic disorders, indicating potential success for this novel approach.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Ahrens-Nicklas, Rebecca Clare — Children's Hosp of Philadelphia
- Study coordinator: Ahrens-Nicklas, Rebecca Clare
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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.