Investigating in utero genome editing for treating genetic diseases
Official title: IN UTERO SMALL AND LARGE ANIMAL RESOURCE CORE
Funded at Children's Hosp of Philadelphia · NIH-10896259 · Data updated 2026-11-12
This study is exploring a way to use gene editing before birth to help prevent serious genetic diseases in babies, and it's designed for families who might be affected by these 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 | 2024 |
| Award (this year) | $315,720 |
| NIH project number | 5U19NS132301-02 |
| Project ID | NIH-10896259 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are expectant parents with a family history of monogenic diseases or those identified as carriers of such conditions.
✕Less likely to help
Patients who are not pregnant or whose conditions are not related to monogenic diseases may not benefit from this research.
What this research studies
This research focuses on in utero genome editing (IUGE) to address monogenic diseases that can cause serious health issues before or shortly after birth. By utilizing the unique characteristics of fetuses, such as their small size and immature immune systems, the research aims to safely apply gene editing techniques to prevent disease onset. The project involves rigorous studies using both small and large animal models, including nonhuman primates, to evaluate the effectiveness and safety of these therapies for both the fetus and the mother.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking treatments for genetic diseases that affect infants before birth.
How similar studies have gone: Previous research has shown promise in gene editing approaches for treating genetic disorders, indicating potential success for this novel application.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Flake, Alan W. — Children's Hosp of Philadelphia
- Study coordinator: Flake, Alan W.
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.