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Article

Molecular impact of antisense oligonucleotide therapy in C9orf72-associated ALS

26 Aug 2025

Abstract excerpt

repeat expansion that leads to toxic RNA transcripts and dipeptide repeat proteins (DPRs). A clinical trial using the antisense oligonucleotide (ASO) BIIB078 to target these transcripts was discontinued after failing to provide clinical benefit. Here, we determine the extent of target engagement in the central nervous system (CNS) and elucidate pharmacodynamic cerebrospinal fluid (CSF) biomarkers following...

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PMID: 40865525
DOI: 10.1016/j.cell.2025.07.045

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