Gene therapy to treat a genetic disorder in dogs
Combination Gene Therapy for Treatment of Canine Mucopolysaccharidosis Type I
This study is testing a new gene therapy to help dogs with Canine Mucopolysaccharidosis Type I by delivering special genes that can fix a missing enzyme, aiming to make them feel better and live healthier lives.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hospital of Orange County NIH-funded |
| Lab location | 1 site (Orange, United States) |
| Project ID | NIH-11046559 on NIH RePORTER |
What this research studies
This research focuses on developing a combination gene therapy for treating Canine Mucopolysaccharidosis Type I, a genetic disorder caused by a deficiency in the α-L-iduronidase enzyme. The approach aims to deliver therapeutic genes using adeno-associated viral vectors to restore enzyme function and reduce the harmful accumulation of glycosaminoglycans in affected dogs. By targeting both systemic and central nervous system symptoms, the therapy seeks to improve the quality of life and overall health of dogs suffering from this condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are dogs diagnosed with Mucopolysaccharidosis Type I, particularly those showing symptoms related to the disease.
Not a fit: Dogs that do not have Mucopolysaccharidosis Type I or those with other unrelated health issues may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a more effective treatment option for dogs with Mucopolysaccharidosis Type I, potentially improving their health and longevity.
How similar studies have performed: Previous research has shown promise in using gene therapy for similar genetic disorders, indicating potential for success in this approach.
Where this research is happening
Orange, United States
- Children's Hospital of Orange County — Orange, United States (Active)
Researchers
- Principal investigator: Wang, Raymond Yu Jeang — Children's Hospital of Orange County
- Study coordinator: Wang, Raymond Yu Jeang
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.