Gene therapy for a rare genetic disorder affecting skeletal development
Gene Therapy for MPS IV A in a Novel Porcine Model of the Disease
This study is looking at a rare disease called MPS IV A and is trying to find better treatments by testing two different gene therapy methods in pigs to see which one works best for helping people with this condition.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Recombinetics, INC. NIH-funded |
| Lab location | 1 site (Eagan, UNITED STATES) |
| Project ID | NIH-11007850 on NIH RePORTER |
What this research studies
This research focuses on MPS IV A, a rare lysosomal storage disease caused by a deficiency in the GALNS enzyme. It aims to create a porcine model to better understand the disease and evaluate the effectiveness of gene therapy as a treatment option. The study will compare two gene transfer methods, adeno-associated virus (AAV) and lentiviral vectors, to determine which is more effective in addressing the severe symptoms of the condition. By advancing genetic therapies, the research seeks to provide a more effective treatment for patients suffering from this debilitating disease.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with MPS IV A or those with a family history of the disease.
Not a fit: Patients with other types of lysosomal storage diseases or those without GALNS deficiency may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for patients with MPS IV A, potentially improving their quality of life.
How similar studies have performed: While gene therapy approaches have shown promise in other genetic disorders, this specific application in MPS IV A is novel and has not been extensively tested.
Where this research is happening
Eagan, UNITED STATES
- Recombinetics, INC. — Eagan, United States (Active)
Researchers
- Principal investigator: Campbell, Jarryd Matthew — Recombinetics, INC.
- Study coordinator: Campbell, Jarryd Matthew
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.