Gene editing to fix blood vessel problems in the brain
Official title: In vivo genome editing for cerebrovascular disorders
Funded at Massachusetts General Hospital · NIH-11448149 · Data updated 2026-11-12
This project tests a gene-editing approach to repair blood vessel walls in a mouse model of a human brain vessel disease, with potential to benefit people living with cerebral small vessel disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | August 26, 2026 |
| Project end | May 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $616,126 |
| NIH project number | 1R01NS147187-01A1 |
| Project ID | NIH-11448149 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with or at risk for cerebral small vessel disease due to ACTA2-related vascular abnormalities or similar small vessel pathologies.
✕Less likely to help
Patients without cerebral small vessel disease or without the ACTA2-associated vascular issue may not benefit from this research.
What this research studies
Researchers will use a mouse model carrying a human disease-causing ACTA2 mutation to study whether correcting the gene early in life can restore artery flexibility, improve blood flow, and protect brain tissues. They will compare early versus later gene editing to see when it works best, and they will measure changes in blood vessels, brain imaging markers, and behavior. The work combines advanced gene-editing techniques with high-resolution brain imaging to understand how actin dysfunction leads to small vessel disease. Results could inform when and how similar edits might be used in people who have this vascular condition.
Why it matters
Potential benefit: If successful, this approach could pave the way for therapies that restore healthy brain blood vessels and prevent stroke or dementia in patients with cerebral small vessel disease.
How similar studies have gone: Similar gene-editing approaches have shown promise in animal models to correct vascular defects and restore vessel function, but human relevance remains to be proven.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Chung, David Young — Massachusetts General Hospital
- Study coordinator: Chung, David Young
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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.