Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Antisense oligonucleotides: rising stars in eliminating RNA toxicity in myotonic dystrophy.

Human gene therapy - 1 May 2013

Gao Zhihua, Cooper Thomas A

Abstract excerpt

Myotonic dystrophy (DM) is a dominantly inherited, multisystemic disease caused by expanded CTG (type 1, DM1) or CCTG (type 2, DM2) repeats in untranslated regions of the mutated genes. Pathogenesis results from expression of RNAs from the mutated alleles that are toxic because of the expanded CUG or CCUG repeats. Increased understanding of the repeat-containing RNA (C/CUG(exp) RNA)-induced toxicity has led to...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Alleles
Share to Cell Nucleus
Share to Genetic Therapy

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Mutation

Share to Myotonic Dystrophy

Share to Oligonucleotides, Antisense

Share to RNA, Messenger

Share to Trinucleotide Repeat Expansion

Source

Open in PubMed Open full text

Identifiers

PMID: 23252746
DOI: 10.1089/hum.2012.212
PMCID: PMC3655630

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.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Antisense oligonucleotides: rising stars in eliminating RNA toxicity in myotonic dystrophy.PMID 23252746Read ↗

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy.Semantic proximity Read ↗

Targeting nuclear RNA for in vivo correction of myotonic dystrophy.Semantic proximity Read ↗

Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseasesSemantic proximity Read ↗

Systemic therapy in an RNA toxicity mouse model with an antisense oligonucleotide therapy targeting a non-CUG sequence within the DMPK 3′UTR RNASemantic proximity Read ↗

Design and Analysis of Effects of Triplet Repeat Oligonucleotides in Cell Models for Myotonic DystrophySemantic proximity Read ↗

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular DiseasesSemantic proximity Read ↗

Therapeutics development in myotonic dystrophy type 1Semantic proximity Read ↗

Antisense oligonucleotides and their applications in rare neurological diseasesSemantic proximity Read ↗

Small Molecules Which Improve Pathogenesis of Myotonic Dystrophy Type 1Semantic proximity Read ↗

AON-induced splice-switching and DMPK pre-mRNA degradation as potential therapeutic approaches for Myotonic Dystrophy type 1Semantic proximity Read ↗

Myotonic dystrophy: approach to therapy.Semantic proximity Read ↗

Perspectives on gene therapy in myotonic dystrophy type 1.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.