Finding the Best Time to Restore Ovarian Function in Galactosemia
Optimal Window of Opportunity for Granulosa Cell Gene Therapy in Galactosemia
['FUNDING_R21'] · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · NIH-11175963
This research aims to find the best way and time to prevent fertility problems in young women with Classic Galactosemia.
Quick facts
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH (nih funded) |
| Locations | 1 site (SALT LAKE CITY, UNITED STATES) |
| Trial ID | NIH-11175963 on ClinicalTrials.gov |
What this research studies
Women with Classic Galactosemia often face ovarian damage and fertility issues, and currently, there are no good treatments to prevent this. This project is developing a new type of gene therapy that uses tiny particles to deliver genetic material directly to the ovary. The goal is to restore the missing GALT activity in ovarian cells, which could help protect their reproductive health. Researchers will also determine the ideal time during development to apply this treatment for the best chance of success.
Who could benefit from this research
Good fit: This research is ultimately for young women and adolescents diagnosed with Classic Galactosemia who are at risk for ovarian damage and fertility issues.
Not a fit: Patients without Classic Galactosemia or those who have already experienced irreversible ovarian damage may not directly benefit from this specific approach.
Why it matters
Potential benefit: If successful, this work could lead to a treatment that prevents fertility impairment in women with Classic Galactosemia.
How similar studies have performed: While insights into Galactosemia and new technologies are emerging, this specific non-viral gene therapy approach targeting ovarian function is novel and being explored in this pilot grant.
Where this research is happening
SALT LAKE CITY, UNITED STATES
- UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH — SALT LAKE CITY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LAI, KENT — UTAH STATE HIGHER EDUCATION SYSTEM--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 below.