Investigating in utero genome editing for treating genetic diseases
IN UTERO SMALL AND LARGE ANIMAL RESOURCE CORE
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 ID | NIH-10896259 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are expectant parents with a family history of monogenic diseases or those identified as carriers of such conditions.
Not a fit: Patients who are not pregnant or whose conditions are not related to monogenic diseases may not benefit from this research.
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 performed: 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
Philadelphia, United States
- 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 below.