Combined AAV gene therapy for dogs with MPS I
Combination Gene Therapy for Treatment of Canine Mucopolysaccharidosis Type I
Researchers are giving a combination AAV-based gene therapy to dogs with MPS I to try to restore the missing IDUA enzyme throughout the body and brain.
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-11289354 on NIH RePORTER |
What this research studies
If your dog has MPS I, this work uses two complementary AAV gene approaches to deliver the missing IDUA enzyme to both the brain and body using a canine model. Scientists will treat affected dogs, then track enzyme activity, levels of stored sugars (GAGs), mobility, heart and spine health, and overall safety over time. The goal is to find a dosing and delivery approach that reduces disease signs without the repeated treatments or risks of current options. Results in dogs will help guide safer, more effective treatments for people with MPS I in the future.
Who could benefit from this research
Good fit: Ideal candidates are young dogs diagnosed with MPS I (α-L-iduronidase deficiency), especially those treated before severe, irreversible neurological or organ damage occurs.
Not a fit: Dogs without MPS I or those with very advanced, irreversible organ or brain injury are unlikely to benefit from this therapy.
Why it matters
Potential benefit: If successful, this approach could provide long-lasting enzyme replacement that reduces organ and neurological damage and improves quality of life.
How similar studies have performed: Prior AAV gene therapy studies in animals have shown promise at lowering disease markers and improving outcomes, but combining systemic and CNS-directed approaches is relatively new.
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.