Editing genes to treat metabolic diseases before and after birth
Official title: Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases
Funded at Children's Hosp of Philadelphia · NIH-10896255 · Data updated 2026-11-12
This study is exploring new ways to use gene editing to help people with metabolic diseases, both before they are born and after, by fixing harmful genes with a special technology called CRISPR.
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 | 2024 |
| Award (this year) | $5,051,652 |
| NIH project number | 5U19NS132301-02 |
| Project ID | NIH-10896255 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals with known genetic metabolic disorders, as well as pregnant women whose fetuses may be at risk for such conditions.
✕Less likely to help
Patients with metabolic diseases that are not caused by genetic mutations or those who are not pregnant may not benefit from this research.
What this research studies
This research focuses on developing innovative gene editing techniques to treat metabolic diseases both before and after birth. Using advanced CRISPR technology, the team aims to deliver precise genetic modifications to patients, potentially correcting harmful mutations that lead to these diseases. The approach includes both in vivo editing, which targets cells within the body, and in utero editing, which could prevent genetic disorders from manifesting in newborns. By utilizing adeno-associated virus (AAV) vectors for delivery, the research seeks to overcome previous challenges in effectively administering gene therapies.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments that prevent or significantly reduce the impact of metabolic diseases in affected individuals.
How similar studies have gone: While gene editing has shown promise in treating certain conditions, this specific approach of in utero editing for metabolic diseases is still largely untested and represents a novel frontier in the field.
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.
Related research
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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.