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Article

Targeting nuclear RNA for in vivo correction of myotonic dystrophy.

Nature - 2 Aug 2012

Wheeler Thurman M, Leger Andrew J, Pandey Sanjay K, MacLeod A Robert, Nakamori Masayuki, Cheng Seng H, Wentworth Bruce M, Bennett C Frank, Thornton Charles A

Abstract excerpt

Antisense oligonucleotides (ASOs) hold promise for gene-specific knockdown in diseases that involve RNA or protein gain-of-function effects. In the hereditary degenerative disease myotonic dystrophy type 1 (DM1), transcripts from the mutant allele contain an expanded CUG repeat and are retained in the nucleus. The mutant RNA exerts a toxic gain-of-function effect, making it an appropriate target for therapeutic...

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Share to Mice, Inbred mdx
Share to Myotonic Dystrophy

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Myotonin-Protein Kinase

Share to Oligonucleotides, Antisense

Share to Ribonuclease H

Share to RNA, Long Noncoding

Share to RNA, Untranslated

Share to Trinucleotide Repeat Expansion

Source

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Identifiers

PMID: 22859208
DOI: 10.1038/nature11362
PMCID: PMC4221572

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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