Gene editing for metabolic liver diseases in monkeys
Official title: Replication study of in vivo postnatal gene editing for metabolic liver disease in the nonhuman primate model
Funded at Children's Hosp of Philadelphia · NIH-11199837 · Data updated 2026-11-12
This study is testing a new way to treat metabolic liver diseases like PKU and HT1 using tiny particles to deliver gene-editing tools, and it's being done in monkeys to see if it can safely improve their liver function before helping people in the future.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | August 1, 2023 |
| Project end | July 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $29,667 |
| NIH project number | 3U19NS132301-02S1 |
| Project ID | NIH-11199837 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for potential benefit from this research would be individuals diagnosed with phenylketonuria (PKU) or hereditary tyrosinemia type 1 (HT1).
✕Less likely to help
Patients with metabolic liver diseases not targeted by this research or those who do not meet specific genetic criteria may not benefit.
What this research studies
This research focuses on developing gene-editing therapies for metabolic liver diseases such as phenylketonuria (PKU) and hereditary tyrosinemia type 1 (HT1) using lipid nanoparticles to deliver the treatment. The approach involves administering a specific mRNA and guide RNA to nonhuman primates to edit genes associated with these conditions. The study aims to replicate previous successful results in juvenile cynomolgus monkeys, where significant gene editing was achieved without adverse effects on liver function. This research could pave the way for future clinical applications in humans.
Why it matters
Potential benefit: If successful, this research could lead to effective gene therapies for patients suffering from metabolic liver diseases.
How similar studies have gone: Previous studies have shown promising results with similar gene-editing approaches, indicating potential for success in this replication study.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Peranteau, William H. — Children's Hosp of Philadelphia
- Study coordinator: Peranteau, William 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.