Improving drug delivery to the brain for lysosomal storage disorders
Official title: Advancing CNS drug delivery via epigenetic modulation
Funded at Cincinnati Childrens Hosp Med Ctr · NIH-10994150 · Data updated 2026-11-12
This study is exploring a new way to help deliver important enzymes to the brain for people with lysosomal storage disorders, using a special technique that could improve treatment and help with neurological issues.
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 | January 18, 2023 |
| Project end | December 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $511,676 |
| NIH project number | 5R01NS132504-03 |
| Project ID | NIH-10994150 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults with lysosomal storage disorders, especially those who have neurological involvement.
✕Less likely to help
Patients with lysosomal storage disorders who do not have neurological symptoms may not receive direct benefits from this research.
What this research studies
This research focuses on enhancing the delivery of essential enzymes to the central nervous system (CNS) for patients with lysosomal storage disorders (LSDs). It investigates the role of a specific receptor, M6PR, which is crucial for transporting these enzymes across the blood-brain barrier. By utilizing a novel approach involving epigenetic modulation through microRNA-143, the study aims to improve enzyme delivery and potentially reverse neurological complications associated with LSDs. Patients may benefit from this innovative method that addresses a significant limitation in current treatment options.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for patients with lysosomal storage disorders, particularly those experiencing neurological symptoms.
How similar studies have gone: Other research has shown promise in using epigenetic modulation for improving drug delivery, indicating potential success for this approach.
Where this research is happening
-
Cincinnati Childrens Hosp Med Ctr
Cincinnati, United States Active
Researchers
- Principal investigator: Pan, Dao — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Pan, Dao
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.