Article

A single-cell map of antisense oligonucleotide activity in the brain

2023-02-14

Abstract excerpt

<h4>ABSTRACT</h4> Antisense oligonucleotides (ASOs) dosed into cerebrospinal fluid (CSF) distribute broadly throughout the brain and hold the promise of treating myriad brain diseases by modulating RNA. CNS tissue is not routinely biopsied in living individuals, leading to reliance on CSF biomarkers to inform on drug target engagement. Animal models can link CSF biomarkers to brain parenchyma, but our understandi...

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Literature Corpus work: 58e5d290-94ca-5e1d-b3bb-7dce2decd5a1
DOI: 10.1101/2023.02.14.528473

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