A novel antibody to treat SOD1-related amyotrophic lateral sclerosis

• Type of Material: Article
• Authors: Nicholas Boulis, Emory University; Anthony Donsante, Emory University
• Language: English
• Date: 2023-06-08
• Publisher: Emory University Libraries
• Publication Version: Final Published Version
• Copyright Statement: © 2023 The Author(s)
• License: Creative Commons Attribution-NoDerivs 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1016/j.omtm.2023.03.011
• Title of Journal or Parent Work: Molecular Therapy - Methods and Clinical Development
• Volume: 29
• Start Page: 236
• End Page: 237
• Research Categories: Health Sciences, Medicine and Surgery

Abstract

The use of antibodies targeting misfolded proteins is a promising approach for the treatment of amyotrophic lateral sclerosis (ALS). In the current issue of Molecular Therapy – Methods and Clinical Development, Minamiyama et al. develop a new monoclonal antibody, D3-1, which appears to bind to a greater variety of superoxide dismutase 1 (SOD1) mutants than previously developed antibodies.1 Intrathecal delivery of the antibody or a cell line expressing a single-chain variable fragment (scFv) of the antibody was sufficient to slow disease progression in a mouse model of ALS.