FUS for therapy of Neuromyelitis Optica – an inflammatory demyelinating disease of the central nervous system

Project Name: FUS for therapy of Neuromyelitis Optica – an inflammatory demyelinating disease of the central nervous system
Status: Active
Primary Site: University of California, San Francisco
Sponsor Organization: Focused Ultrasound Foundation
Funded Date: Mar 25, 2019
Funding Program: Research Award
Funding Period: Mar 25, 2019 - Mar 24, 2020
Amount Awarded: $100,000.00

Abstract

This project will advance antibody and cell therapeutics for neuromyelitis optica, an autoimmune inflammatory disease of the central nervous system associated with demyelination and neurological impairment. The disease is caused by autoantibodies that bind to astrocyte protein aquaporin-4. Focused ultrasound will be used to transiently open the blood-brain barrier to deliver antibody and cell therapeutics, including a therapeutic antibody (aquaporumab) that blocks autoantibody binding to aquaporin-4, and stem cells that promote remyelination. The methods established in this project may be widely applicable to delivery of large molecule and cell therapeutics into the central nervous system for treatment of neurological diseases

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