Creating a large animal model to study a rare neurological disorder called sialidosis
Official title: Generation of a Large Animal Model of Sialidosis to Enable Future Translation of Novel Therapeutics
Funded at Univ of Massachusetts Med Sch Worcester · NIH-10578286 · Data updated 2026-11-12
This study is creating specially designed sheep to help us learn more about sialidosis, a rare and serious brain disorder, so we can test new treatments before using them in people.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | January 1, 2023 |
| Project end | June 30, 2025 |
| Funding year | 2023 |
| Award (this year) | $505,640 |
| NIH project number | 1R61NS123756-01A1 |
| Project ID | NIH-10578286 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with sialidosis or those who are carriers of the NEU1 gene mutation.
✕Less likely to help
Patients with other neurological disorders unrelated to sialidosis may not benefit from this research.
What this research studies
This research focuses on developing a large animal model to better understand sialidosis, a rare and fatal neurological disorder caused by a genetic mutation. The team will use advanced gene editing techniques to create sheep that mimic the human condition of sialidosis. By studying these animals, researchers aim to evaluate how well they replicate the symptoms and biological characteristics of the disease. This model will help in testing potential gene therapies before they are applied to human patients.
Why it matters
Potential benefit: If successful, this research could lead to effective gene therapies for patients suffering from sialidosis.
How similar studies have gone: Previous research has shown success in using large animal models to study human genetic diseases, making this approach promising.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Gray-Edwards, Heather L — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Gray-Edwards, Heather L
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.