Antibody targeting TDP-43 mitigates pathogenic pathways induced by the cerebrospinal fluid of ALS.

ABSTRACT: Amyotrophic lateral sclerosis (ALS) is an incurable neurodegenerative disease characterized by the cytoplasmic mislocalization and accumulation of TAR DNA binding protein 43 (TDP-43). We reported previously the protective effects in a transgenic mouse model expressing ALS-linked mutant TDP-43^A315T of a monoclonal antibody, called E6, binding specifically to the RNA Recognition Motif 1 (RRM1) domain of TDP-43. Here, we tested the effects of E6 antibody in an animal model of sporadic ALS based on the intracerebroventricular (i.c.v.) infusion during 14 days of cerebrospinal fluid (CSF) from sporadic ALS patients into transgenic mice expressing human TDP-43^WT. Either intrathecal (i.t.) or i.c.v. injection of E6 antibody conferred protective effects in this model of diseas

SUBMITTER: Poulin-Briere A 

PROVIDER: S-EPMC12664456 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature