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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