A human-derived antibody targets misfolded SOD1 and ameliorates motor symptoms in mouse models of amyotrophic lateral sclerosis

Authors: Marcel Maier, Tobias Welt, Fabian Wirth, Fabio Montrasio, Daniel F. Preisig, Jordan McAfoose, Fernando G. Vieira, Luka Kulic, Claudia Späni, Thilo Stehle, Steve Perrin, Markus Weber, Christoph Höck, Roger M. Nitsch, Jan Grimm - Science Translational Medicine 2018 cited by 90

mutations, treatment with the α-miSOD1 antibody delayed the onset of motor symptoms, extended survival by up to 2 months, and reduced aggregation of misfolded SOD1 and motor neuron degeneration. These effects were obtained whether α-miSOD1 antibody treatment was administered by direct brain infusion or peripheral administration. These results support the further development of α-miSOD1 antibody as a candidate treatment for ALS involving misfolding of SOD1.