Delivering enzymes to the brain to treat Gaucher disease
Official title: Systemic Enzyme Delivery by a Brain Targeted SapC-DOPS Nanocarrier for Treatment of Neuronopathic Gaucher Disease
Funded at Cincinnati Childrens Hosp Med Ctr · NIH-10941209 · Data updated 2026-11-12
This study is looking at a new way to send a helpful enzyme directly to the brain for people with neuronopathic Gaucher disease, hoping to ease their symptoms by reducing harmful substances that build up in the brain.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project start | July 1, 2024 |
| Project end | June 30, 2029 |
| Funding year | 2024 |
| Award (this year) | $653,309 |
| NIH project number | 1R01NS138309-01 |
| Project ID | NIH-10941209 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with neuronopathic Gaucher disease, particularly those experiencing neurological symptoms.
✕Less likely to help
Patients with non-neuronopathic forms of Gaucher disease or those who do not have a confirmed diagnosis may not benefit from this research.
What this research studies
This research focuses on developing a new method to deliver enzymes directly to the brain for patients with neuronopathic Gaucher disease, a severe form of the condition that affects the central nervous system. The approach utilizes a specialized nanocarrier, SapC-DOPS, which can cross the blood-brain barrier and transport a stable enzyme, fGCase, effectively into brain tissues. By improving the delivery and stability of the enzyme, the research aims to reduce harmful glycolipid accumulation in the brain, potentially alleviating neurological symptoms associated with the disease.
Why it matters
Potential benefit: If successful, this research could significantly improve treatment outcomes for patients with neuronopathic Gaucher disease by providing a more effective enzyme replacement therapy.
How similar studies have gone: Previous research has shown promise in using nanocarrier systems for drug delivery across the blood-brain barrier, indicating potential success for this novel approach.
Where this research is happening
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Cincinnati Childrens Hosp Med Ctr
Cincinnati, United States Active
Researchers
- Principal investigator: Sun, Ying — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Sun, Ying
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.