A cell-penetrant peptide blocking C9ORF72-repeat RNA nuclear export reduces the neurotoxic effects of dipeptide repeat proteins.
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Sci Transl Med. 2023 Mar;15(685):eabo3823. doi: 10.1126/scitranslmed.abo3823. Epub 2023 Mar 1.
Sci Transl Med. 2023.
PMID: 36857431