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Article

A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground.

Archives of neurology - 1 Jan 2009

Yokota Toshifumi, Takeda Shin'ichi, Lu Qi-Long, Partridge Terence A, Nakamura Akinori, Hoffman Eric P

Abstract excerpt

Antisense oligonucleotides are short nucleic acid sequences designed for use as small-molecule drugs. They recognize and bind to specific messenger RNA (mRNA) or pre-mRNA sequences to create small double-stranded regions of the target mRNA that alter mRNA splicing patterns or inhibit protein translation. Antisense approaches have been actively pursued as a form of molecular medicine for more than 20 years, but...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 19139297
DOI: 10.1001/archneurol.2008.540
PMCID: PMC4111150

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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