Focused Ultrasound Treatment of Cerebral Cavernous Malformations

Project Name: Focused Ultrasound Treatment of Cerebral Cavernous Malformations
Status: Active
Primary Site: University of Virginia Health System
Sponsor Organization: Focused Ultrasound Foundation
Funded Date: May 05, 2020
Funding Period: May 05, 2020 - May 05, 2021
Funding Program: Brain Program
Category: Pre-Clinical
Amount Awarded: $99,944.75


The objective of the proposed research is to utilize Focused Ultrasound (FUS) to target and treat cerebral cavernous malformations (CCM) in a mouse model of the disease. To accomplish this, we will generate mice harboring CCM by conditional deletion of the genes responsible for the pathology (Krit1/Ccm1 and Ccm2/Malcavernin) in endothelial cells, and utilize FUS in conjunction with 5-Aminolevulinic acid (5-ALA) to target the malformations. If FUS can be safely used to target the vascular lesions, and if the treatment proves efficacious, it would provide a minimally invasive modality for treatment of a common and devastating disease in the young.

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