Research ArticleALS

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

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Science Translational Medicine  05 Dec 2018:
Vol. 10, Issue 470, eaah3924
DOI: 10.1126/scitranslmed.aah3924

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Wellderly antibodies to combat ALS

Amyotrophic lateral sclerosis (ALS) is characterized by the presence of abnormal forms of proteins such as superoxide dismutase 1 (SOD1) that accumulate in neurons, which then degenerate and die. Maier et al. have now developed a human antibody derived from a cohort of healthy elderly individuals that specifically recognizes abnormal SOD1 in postmortem spinal cord tissue from patients with ALS. When administered to animal models of ALS, the antibody delayed the onset of motor impairments, extended the survival of the animals, and reduced the degeneration of neurons and accumulation of SOD1 aggregates. These results support the further evaluation of this human antibody for the potential treatment of ALS involving misfolding of SOD1.