Using nanoparticles to deliver enzyme therapy for brain disorders
Official title: Nanoparticle Distributed Intravenous Enzyme Replacement Therapy (NanoDIVERT)
Funded at Clemson University · NIH-10723846 · Data updated 2026-11-12
This study is testing a new way to help people with GM1 gangliosidosis by using tiny particles to deliver an important enzyme directly to the brain, aiming to improve treatment options for those who currently have few choices.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Clemson University NIH-funded |
| Lab location | 1 site (Clemson, United States) |
| Project start | July 1, 2023 |
| Project end | June 30, 2025 |
| Funding year | 2023 |
| Award (this year) | $404,643 |
| NIH project number | 1R21NS133477-01 |
| Project ID | NIH-10723846 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with GM1 gangliosidosis or other lysosomal storage disorders that impact the central nervous system.
✕Less likely to help
Patients with lysosomal storage disorders that do not affect the brain or those who do not have a genetic basis for their condition may not benefit from this research.
What this research studies
This research investigates a novel approach to enzyme replacement therapy (ERT) for patients with lysosomal storage disorders that affect the brain, specifically GM1 gangliosidosis. The study utilizes specially designed nanoparticles, called polymersomes, to encapsulate the enzyme β-galactosidase and facilitate its delivery across the blood-brain barrier, which typically prevents such treatments from reaching the central nervous system. By administering these nanoparticles intravenously, the goal is to improve the therapeutic outcomes for patients who currently have limited treatment options due to the challenges posed by the blood-brain barrier. Preliminary results in animal models suggest that this method can effectively transport the enzyme to the brain, potentially offering a new hope for affected individuals.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with GM1 gangliosidosis and similar disorders, improving their quality of life and extending survival.
How similar studies have gone: While enzyme replacement therapy has been successful for some lysosomal storage disorders, the use of nanoparticles to cross the blood-brain barrier is a novel approach that has shown promising preliminary results but is still largely untested in humans.
Where this research is happening
-
Clemson University
Clemson, United States Active
Researchers
- Principal investigator: Larsen, Jessica Marie — Clemson University
- Study coordinator: Larsen, Jessica Marie
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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.