Article

Comprehensive preclinical evaluation of human-derived anti-poly-GA antibodies in cellular and animal models of C9ORF72 disease

2022-01-15

Abstract excerpt

Hexanucleotide G 4 C 2 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 show toxic effects in vivo as well as in vitro and are key targets for therapeutic intervention. We generated human antibodies that bind DPRs with high affinit...

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Literature Corpus work: 3cb1d246-945d-5563-a257-9c4298cff745
DOI: 10.1101/2022.01.13.475329

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