In utero gene-editing resource for animal models
IN UTERO SMALL AND LARGE ANIMAL RESOURCE CORE
This project builds animal tests and expertise to help develop gene-editing treatments given before birth for babies with serious inherited genetic disorders.
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-11145247 on NIH RePORTER |
What this research studies
This resource core supports rigorous animal work in mice and large animals, including nonhuman primates, to model gene-editing therapies delivered before birth. Researchers will develop methods for delivering gene editors to the fetus, track where the treatment goes in the body, and check both fetal and maternal safety. The core provides technical services, standardized protocols, and data analysis to help investigators design studies that could support future human trials. While the work is preclinical, it aims to speed safe translation of prenatal gene therapies for families facing early-onset monogenic diseases.
Who could benefit from this research
Good fit: Although this grant funds animal research rather than human enrollment, eventual trial candidates would be pregnant people carrying fetuses diagnosed with severe, early-onset monogenic disorders.
Not a fit: People with adult-onset genetic conditions, non-genetic diseases, or those without a prenatal diagnosis are unlikely to benefit from these in utero approaches.
Why it matters
Potential benefit: If successful, this work could enable safe prenatal gene therapies that prevent or reduce severe damage from genetic diseases before babies are born.
How similar studies have performed: Early proof-of-concept studies in mice and limited large-animal work have shown promise, but clinical use of in utero gene editing in humans remains experimental.
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.