Improving ovarian function in girls with Classic Galactosemia through gene therapy
Official title: Optimal Window of Opportunity for Granulosa Cell Gene Therapy in Galactosemia
Funded at University of Utah · NIH-10787066 · Data updated 2026-11-12
This study is exploring a new gene therapy to help young women with Classic Galactosemia improve their ovarian function and fertility by delivering a special treatment directly to their ovaries, with the goal of preventing damage and enhancing their chances of having children.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Utah NIH-funded |
| Lab location | 1 site (Salt Lake City, United States) |
| Project start | August 23, 2024 |
| Project end | July 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $253,167 |
| NIH project number | 1R21HD113931-01 |
| Project ID | NIH-10787066 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are young women aged 12-20 who have been diagnosed with Classic Galactosemia and are experiencing fertility impairments.
✕Less likely to help
Patients who do not have Classic Galactosemia or who are outside the age range of 12-20 may not benefit from this research.
What this research studies
This research investigates a novel gene therapy approach aimed at restoring ovarian function in young women affected by Classic Galactosemia, a genetic disorder that can lead to severe fertility issues. The study will utilize a non-viral nucleic acid therapy that targets granulosa cells in the ovaries, aiming to deliver a gene that can correct the underlying deficiency. By determining the optimal timing for treatment, the researchers hope to prevent ovarian damage and improve reproductive health outcomes for affected individuals.
Why it matters
Potential benefit: If successful, this research could significantly improve fertility and overall reproductive health for young women with Classic Galactosemia.
How similar studies have gone: While gene therapy for other conditions has shown promise, this specific approach targeting ovarian function in Classic Galactosemia is novel and has not been extensively tested.
Where this research is happening
-
University of Utah
Salt Lake City, United States Active
Researchers
- Principal investigator: Lai, Kent — University of Utah
- Study coordinator: Lai, Kent
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.