Precise gene editing before or after birth for metabolic diseases
Official title: Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases
Funded at Children's Hosp of Philadelphia · NIH-11145243 · Data updated 2026-11-12
This project develops precise gene-editing treatments given before birth or soon after to correct genetic metabolic conditions in children.
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) | $5,059,052 |
| NIH project number | 5U19NS132301-03 |
| Project ID | NIH-11145243 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be fetuses or newborns diagnosed with specific single-gene metabolic disorders that are correctable by an adenine base editor or similar precise edit.
✕Less likely to help
Patients whose conditions are not caused by the specific single-base mutations targeted, who have irreversible organ damage, or whose disease mechanism is not addressable by base editing are unlikely to benefit.
What this research studies
If you or your baby has a genetic metabolic disease, this project is developing precise "base editing" treatments that can change single DNA letters to fix disease-causing genes. Researchers are testing delivery methods such as AAV viral carriers and lipid nanoparticles, and trying treatments given before birth (in utero) as well as after birth to reach organs like the liver. Most work is being done in animal models now to study safety, immune reactions, and how well the edits last, with the goal of moving the best approaches toward human use. The team at Children's Hospital of Philadelphia aims to identify which conditions and timing offer the best chance of a lasting benefit with acceptable risks.
Why it matters
Potential benefit: If successful, this work could permanently correct the underlying genetic cause of some metabolic diseases when treated before or shortly after birth, reducing long-term illness or preventing it entirely.
How similar studies have gone: Related in vivo genome-editing approaches have shown promising results in animal models and early liver-targeted human work (e.g., TTR, PCSK9, ANGPTL3), but prenatal in utero base editing remains experimental.
Where this research is happening
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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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Postnatal and Prenatal Base Editing Therapies for Metabolic Diseases
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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.