Postnatal and Prenatal Base Editing Therapies for Metabolic Diseases
Official title: Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases
Funded at Children's Hosp of Philadelphia · NIH-11421631 · Data updated 2026-11-12
This grant aims to advance genetic medicine that could one day treat metabolic diseases before or after birth using precise genome editing techniques.
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) | $5,455,342 |
| NIH project number | 3U19NS132301-04S1 |
| Project ID | NIH-11421631 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are infants or fetuses diagnosed with genetic metabolic diseases that could be addressed by genome-editing therapies in development.
✕Less likely to help
Patients without a genetic metabolic condition targeted by the specific base-editing approaches being developed may not benefit.
What this research studies
This program explores how to safely deliver and use base-editing tools to correct genetic problems in living organisms, including efforts to reach fetuses and newborns. It builds on recent successes with CAR-T therapies and blood disease treatments to apply genome editing directly in the body. The work includes studying delivery methods, safety, and effectiveness in animal models as a step toward human trials. The project is centered at Children’s Hospital of Philadelphia, led by Dr. William H. Peranteau, with a focus on prenatal and postnatal therapeutic strategies.
Why it matters
Potential benefit: If successful, it could lead to new treatments that correct genetic causes of metabolic diseases before or shortly after birth, potentially reducing disease burden and improving outcomes.
How similar studies have gone: Previous work shows promise in ex vivo genome editing and limited in vivo attempts, but in utero and somatic editing in humans remains early and experimental.
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 for Metabolic Diseases
A large NIH grant is funding research to develop gene-editing methods that could treat metabolic diseases before and after birth.
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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.