How tiny cell vesicles may cause brain cavernous malformations
Investigating the intracellular vesicle-mediated mechanism contributing to cerebral cavernous malformation
['FUNDING_R01'] · UNIVERSITY OF VIRGINIA · NIH-11483416
This project looks at how changes inside blood-vessel cells in the brain lead to cerebral cavernous malformations, with special attention to people who have CCM3 gene defects.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF VIRGINIA (nih funded) |
| Locations | 1 site (CHARLOTTESVILLE, UNITED STATES) |
| Trial ID | NIH-11483416 on ClinicalTrials.gov |
What this research studies
As a patient, I would learn that researchers are focusing on the CCM3 (Pdcd10) gene because losing it produces more severe brain lesions. They use a specialized mouse model that develops brain vessel lesions in adulthood so they can watch lesion formation live. The team is studying tiny vesicles called caveolae and proteins like caveolin-1 and Angiopoietin-2 to understand how the blood-brain barrier becomes leaky. By defining these steps and testing treatments in mice, they hope to find targets that could lead to new therapies.
Who could benefit from this research
Good fit: People diagnosed with cerebral cavernous malformations, especially those with known CCM3 (Pdcd10) mutations, would be the most relevant group.
Not a fit: People without CCM or those seeking immediate clinical treatment are unlikely to receive direct benefit from this basic laboratory research in the near term.
Why it matters
Potential benefit: If successful, this work could point to new drugs or approaches to protect the blood-brain barrier and prevent lesion growth or bleeding in people with CCM.
How similar studies have performed: Previous lab studies have identified related pathways (such as Angiopoietin-2 secretion and caveolin-1 changes) and established mouse models, but moving these findings into human treatments is still experimental.
Where this research is happening
CHARLOTTESVILLE, UNITED STATES
- UNIVERSITY OF VIRGINIA — CHARLOTTESVILLE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ZHOU, JENNY HUANJIAO — UNIVERSITY OF VIRGINIA
- Study coordinator: ZHOU, JENNY HUANJIAO
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 below.