Advancing CNS drug delivery through epigenetic tweaks
Official title: Advancing CNS drug delivery via epigenetic modulation
Funded at Cincinnati Childrens Hosp Med Ctr · NIH-11617801 · Data updated 2026-11-12
A medical research project aims to help people with lysosomal storage disorders by improving how treatments reach 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 | January 18, 2023 |
| Project end | December 31, 2026 |
| Funding year | 2026 |
| Award (this year) | $40,544 |
| NIH project number | 3R01NS132504-04S1 |
| Project ID | NIH-11617801 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with lysosomal storage disorders, particularly those with CNS involvement, could benefit from more effective CNS enzyme delivery.
✕Less likely to help
Individuals without CNS involvement or those not eligible for CNS-delivery strategies may see limited benefit.
What this research studies
This project looks at making brain therapies for inherited storage disorders work better by changing epigenetic signals that control a receptor needed to deliver enzymes to the brain. Researchers use a dual luciferase system and mutations to study microRNA miR-143’s effect on M6PR levels at brain blood vessels in mice, with a goal that enhanced delivery could improve outcomes. The study is conducted in a Cincinnati Children’s Hospital setting and focuses on translating findings toward better CNS treatment options for patients who currently have limited CNS benefits from existing therapies.
Why it matters
Potential benefit: If successful, this work could lead to more effective brain-directed treatments for lysosomal storage disorders, potentially reducing neurological symptoms.
How similar studies have gone: Previous work shows M6PR and CNS delivery are promising targets, but challenges remain in translating to humans; this study builds on prior mouse-model findings.
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.