Postnatal and Prenatal Base Editing for Metabolic Diseases
Official title: Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases
Funded at Children's Hosp of Philadelphia · NIH-11389343 · Data updated 2026-11-12
A large NIH grant is funding research to develop gene-editing methods that could treat metabolic diseases before and after birth.
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 | 2026 |
| Award (this year) | $9,996,957 |
| NIH project number | 4U19NS132301-04 |
| Project ID | NIH-11389343 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are patients with metabolic diseases caused by genetic defects that could be corrected by genome editing.
✕Less likely to help
Patients without these specific genetic metabolic conditions or those not eligible for gene-delivery approaches may not benefit.
What this research studies
This project aims to advance safe, effective genome editing inside the body to correct genetic problems that cause metabolic diseases. Researchers will explore delivery of base-editing tools via viral vectors, study prenatal and postnatal treatment options, and test in relevant models to understand safety and effectiveness. The focus is on translating genome-editing advances toward real patient care, including therapies that could prevent or lessen disease symptoms from birth onward.
Why it matters
Potential benefit: If successful, this work could lead to new therapies that prevent or lessen metabolic disease burden starting before birth and continue after birth.
How similar studies have gone: Similar in vivo genome-editing approaches in liver have shown progress, supporting potential but with still-significant safety considerations.
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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Postnatal and Prenatal Base Editing Therapies for Metabolic Diseases
This grant aims to advance genetic medicine that could one day treat metabolic diseases before or after birth using precise genome editing techniques.
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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.