Gene therapy for a rare genetic disorder affecting skeletal development
Official title: Gene Therapy for MPS IV A in a Novel Porcine Model of the Disease
Funded at Recombinetics, INC. · NIH-11007850 · Data updated 2026-11-12
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 start | September 18, 2024 |
| Project end | August 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $384,999 |
| NIH project number | 1R43AR084639-01A1 |
| Project ID | NIH-11007850 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with MPS IV A or those with a family history of the disease.
✕Less likely to help
Patients with other types of lysosomal storage diseases or those without GALNS deficiency may not benefit from this research.
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.
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 gone: 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
-
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.