The Scripps Research Institute
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Principal Investigator: Dr. Roberto Baccala Project Title: Proteomic Studies Leading to Development of |
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Neuromyelitis optica (NMO), also known as Devic’s disease, is a chronic inflammatory demyelinating disorder of the central nervous system (CNS) mainly affecting the optic nerves and spinal cord. Because of overlapping clinical characteristics, NMO was frequently misdiagnosed as multiple sclerosis (MS). Accuracy in diagnosis was highly improved by the recent discovery of disease-specific serum autoantibodies (NMO-IgG) with specificity for aquaporin-4 (AQP4), the main water channel in the CNS. Several observations have provided strong indirect evidence that these autoantibodies play a critical role in the physiopathology of NMO. Nevertheless, several important questions remain unresolved, particularly due to the absence of appropriate animal models. The focus of our proposal will be to develop mouse models of AQP4 autoimmunity, drawing from our expertise in autoimmune diseases and mechanisms by which immunological tolerance to self-antigens can be broken. Availability of such models will permit to directly study humoral and cellular contributions to this disease, develop monoclonal antibodies, T cell hybridomas and transgenic mice, and assess novel immunosuppressive therapies for NMO. The Scripps NMO Team
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