Making guide RNAs to support in-body gene editing treatments for metabolic liver diseases
Official title: Guide RNA manufacturing capabilities for platform in vivo base editing therapies for metabolic liver diseases
Funded at Children's Hosp of Philadelphia · NIH-11337355 · Data updated 2026-11-12
This project is developing ways to produce the RNA guides needed for in-body gene-editing treatments aimed at people with inherited metabolic liver conditions.
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) | $1,000,000 |
| NIH project number | 3U19NS132301-03S1 |
| Project ID | NIH-11337355 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for future therapies enabled by this project would be people with inherited metabolic liver diseases or genetic lipid disorders that can be treated by targeted base editing in the liver.
✕Less likely to help
People with non-genetic liver conditions (for example viral hepatitis or alcohol-related liver disease) or diseases not amenable to liver-directed base editing are unlikely to benefit from these specific approaches.
What this research studies
From my perspective as a patient, the team is building better methods to manufacture the short RNA pieces (guide RNAs) that direct base-editing tools to the right spot in liver cells. They are combining these guide RNAs with in-body delivery approaches like viral vectors and lipid nanoparticles and testing them in laboratory and animal models to check safety and precision. The goal is to create a reliable, scalable manufacturing process so future therapies can be made consistently and given to people safely. If this work goes well, it could speed development of single-treatment gene edits for several inherited liver metabolic disorders.
Why it matters
Potential benefit: If successful, this work could make in-body gene-editing treatments for metabolic liver diseases safer, more reliable, and easier to bring into clinical trials and patient care.
How similar studies have gone: Early in vivo base-editing work in animals and a few human/primate examples (for targets like TTR, PCSK9, and ANGPTL3) have shown promise, but scalable guide RNA manufacturing for clinical in-body base editing is still a novel and developing area.
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.
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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.