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Department of Quantitative Biomedicine

The Polymenidou group and collaborators systematically evaluate human-derived anti-poly-GA antibodies in C9orf72 disease models

Comprehensive evaluation of human-derived anti-poly-GA antibodies in cellular and animal models of C9orf72 disease

Hexanucleotide G4C2 repeat expansions in the C9orf72 gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Dipeptide repeat proteins (DPRs) generated by translation of repeat-containing RNAs are key targets for therapeutic intervention. In this work, the Polymenidou group and collaborators generated human antibodies that bind DPRs with high affinity and specificity, systematically characterized these against multiple DPR species and tested the biological effects of antibodies targeting poly-GA in different cellular and mouse models. 

See Jambeau et al, PNAS


Fig 1. chα-GA3 is internalized via vesicular compartments in human neurons, but does not alter GA50-GFP vesicular localization. © 2022 The Authors