Neuromyelitis Optica Spectrum Disorder (NMOSD) is attributed to CNS autoimmune disorder characterized by optic neuritis and acute myelitis with an inflammatory, demyelinating process of the central nerve system. The NMO-IgG has been proven to target a subtype of the water channel protein, known as aquaporin-4 (AQP4) that is expressed in astrocyte foot processes at the blood–brain barrier of CNS. NMO-IgG is known as AQP4 autoantibodies attack the extracellular domains of AQP4 leading to AQP4 loss and astrocytopathy via complement-dependent cytotoxicity (CDC) and antibody-dependent cell-mediated cytotoxicity (ADCC). Herein, we successfully generate the peptide-specific AQP4 monoclonal antibody named AQP001 and prove the potent of AQP001 to compete with NMO-IgG and preserve the intact AQP4 protein from NMO-IgG attack in vitro, implying the therapeutic potential of AQP001 in the human NMOSD in the future.

Shyi-Jou Chen
Tri-Service General Hospital, National Defense Medical Center
Taiwan

Cheih-Min Chen
National Defense Medical Center, Taipei, Taiwan
Taiwan

Jim-Wen Chen
National Defense Medical Center, Taipei, Taiwan
Taiwan

Huey-Kang Sytwu
National Health Research Institutes
Taiwan