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Antisense therapies in neurological diseases

17 Nov 2021

Abstract excerpt

Advances in targeted regulation of gene expression allowed new therapeutic approaches for monogenic neurological diseases. Molecular diagnosis has paved the way to personalized medicine targeting the pathogenic roots: DNA or its RNA transcript. These antisense therapies rely on modified nucleotides sequences (single-strand DNA or RNA, both belonging to the antisense oligonucleotides family, or double-strand...

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PMID: 35286370
DOI: 10.1093/brain/awab423

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