Delivering therapeutic molecules to the brain using engineered exosomes for Huntington's disease
Official title: Brain-targeted delivery of therapeutic molecules by exosomes derived from engineered human iPS cells: a potential therapeutic approach for Huntington's disease
Funded at Johns Hopkins University · NIH-10764280 · Data updated 2026-11-12
This study is exploring a new way to deliver treatments directly to the brain for people with Huntington's disease, using tiny particles from specially engineered cells to help target the harmful protein that causes the condition, with the hope of creating a more effective therapy.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | January 15, 2023 |
| Project end | December 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $204,302 |
| NIH project number | 5R21NS125350-02 |
| Project ID | NIH-10764280 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Huntington's disease who are seeking new therapeutic options.
✕Less likely to help
Patients with other neurological disorders unrelated to Huntington's disease may not benefit from this research.
What this research studies
This research focuses on developing a method to deliver therapeutic molecules directly to the brain for patients with Huntington's disease, a severe neurodegenerative condition. The approach utilizes exosomes derived from engineered human induced pluripotent stem cells (iPSCs), which are tiny vesicles that can cross the blood-brain barrier. By modifying these exosomes to enhance their targeting capabilities, the research aims to improve the delivery of treatments that can suppress the harmful effects of the mutant huntingtin protein. Patients may benefit from a more effective therapy that can reach the affected areas of the brain.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for Huntington's disease by improving the delivery of therapeutic molecules to the brain.
How similar studies have gone: Previous research has shown promise in using exosomes for drug delivery in other contexts, suggesting potential success for this novel approach in Huntington's disease.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Li, Pan — Johns Hopkins University
- Study coordinator: Li, Pan
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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.