Gene therapy for a genetic disorder called classic galactosemia
Official title: Gene Therapy in a GALT-null Rat Model of Classic Galactosemia
Funded at Emory University · NIH-11089113 · Data updated 2026-11-12
This study is exploring a new gene therapy to help treat classic galactosemia, a genetic condition that makes it hard for the body to process a sugar called galactose, by using a special virus to deliver the missing gene to rats that have a similar condition, with hopes that it can prevent serious long-term problems like learning and movement issues.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project start | September 1, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $195,625 |
| NIH project number | 1R56DK137888-01A1 |
| Project ID | NIH-11089113 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with classic galactosemia, particularly children who are affected by this genetic disorder.
✕Less likely to help
Patients who do not have classic galactosemia or those who are not responsive to gene therapy may not benefit from this research.
What this research studies
This research investigates the use of gene therapy to treat classic galactosemia (CG), a serious genetic condition that affects how the body processes galactose. The study will utilize a specific viral vector to deliver the GALT gene, which is missing in patients with CG, to a rat model that mimics the disease. By observing the effects of this therapy, researchers aim to understand if it can prevent long-term complications associated with CG, such as cognitive and motor deficits. This approach could potentially lead to a new treatment option that addresses the root cause of the disease rather than just managing symptoms.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that significantly improves the quality of life for patients with classic galactosemia.
How similar studies have gone: While gene therapy for metabolic disorders is a developing field, this specific approach using AAV9-based GALT gene therapy in classic galactosemia is relatively novel and has not been extensively tested in humans yet.
Where this research is happening
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Emory University
Atlanta, United States Active
Researchers
- Principal investigator: Fridovich-Keil, Judith L. — Emory University
- Study coordinator: Fridovich-Keil, Judith L.
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.