Gene therapy for glycogen storage disease type III
This study is testing a new gene therapy for people with glycogen storage disease type III, aiming to help their bodies break down harmful glycogen buildup in muscles and the liver by using a special virus to deliver a helpful enzyme.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project ID | NIH-11053467 on NIH RePORTER |
What this research studies
This research investigates a new gene therapy approach for glycogen storage disease type III (GSD III), a genetic disorder that leads to harmful glycogen accumulation in muscles and liver. The therapy utilizes an adeno-associated virus (AAV) vector to deliver a modified version of the glycogen debranching enzyme, which is crucial for breaking down glycogen. By incorporating a small bacterial enzyme and a dual promoter to minimize immune response, the goal is to enable long-term expression of the therapeutic enzyme in affected tissues. This innovative approach aims to provide a potential treatment option for patients suffering from this debilitating condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with glycogen storage disease type III, particularly those with both muscle and liver involvement.
Not a fit: Patients with other forms of glycogen storage diseases or those without a confirmed diagnosis of GSD III may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a long-term therapeutic solution for patients with glycogen storage disease type III, potentially improving their quality of life and reducing disease-related complications.
How similar studies have performed: Previous research has shown promise in using AAV-mediated gene therapy for other inherited muscle and liver disorders, indicating potential for success in this novel application.
Where this research is happening
Durham, United States
- Duke University — Durham, United States (Active)
Researchers
- Principal investigator: Sun, Baodong — Duke University
- Study coordinator: Sun, Baodong
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.