Gene therapy for a genetic disorder called classic galactosemia
Gene Therapy in a GALT-null Rat Model of Classic Galactosemia
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 ID | NIH-11089113 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with classic galactosemia, particularly children who are affected by this genetic disorder.
Not a fit: Patients who do not have classic galactosemia or those who are not responsive to gene therapy may not benefit from this research.
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 performed: 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
Atlanta, United States
- 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 below.