Prenatal gene therapy approach to prevent cleft lip and palate in a human-like mouse model
Official title: In utero rescue of cleft lip and palate in a humanized mouse model
Funded at University of Colorado Denver · NIH-11261221 · Data updated 2026-11-12
The team is trying a prenatal gene-delivery approach in humanized mice to prevent cleft lip and palate linked to TFAP2A mutations, with the goal of helping babies born with those conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Colorado Denver NIH-funded |
| Lab location | 1 site (Aurora, UNITED STATES) |
| Project start | April 1, 2023 |
| Project end | January 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $435,364 |
| NIH project number | 5R01DE032740-04 |
| Project ID | NIH-11261221 on NIH RePORTER |
Who could benefit from this research
✓Could help
The work is most relevant to families affected by TFAP2A-related syndromes (such as Branchio-Oculo-Facial Syndrome) or those with a known TFAP2A mutation who might be candidates if human trials are developed.
✕Less likely to help
People whose cleft lip or palate is caused by other genes or non-genetic/environmental factors would be less likely to benefit from a TFAP2A-targeted prenatal approach.
What this research studies
Researchers are using mice engineered to mimic human TFAP2A-related cleft lip and palate to test a prenatal corrective therapy. They deliver a gene-carrying AAV vector into the developing embryo before birth and follow facial development to see if clefts are prevented. The team examines both anatomical outcomes and safety signals in treated animals. Findings will guide whether this approach could move toward future prenatal treatments for at-risk pregnancies.
Why it matters
Potential benefit: If successful, this could point toward prenatal treatments that prevent or reduce cleft lip and palate in babies with TFAP2A-related conditions.
How similar studies have gone: Some prenatal gene-delivery approaches have corrected defects in animal models, but in utero rescue specifically for TFAP2A-linked craniofacial clefts is largely novel.
Where this research is happening
-
University of Colorado Denver
Aurora, United States Active
Researchers
- Principal investigator: Williams, Trevor J — University of Colorado Denver
- Study coordinator: Williams, Trevor J
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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.