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Article

Perspectives on gene therapy in myotonic dystrophy type 1.

Journal of neuroscience research - 1 Mar 2011

Magaña Jonathan J, Cisneros Bulmaro

Abstract excerpt

Myotonic dystrophy type 1 (DM1) is an autosomal dominant neuromuscular disorder caused by a CTG expansion mutation located in the 3' untranslated region of the DMPK (DM1 protein kinase) gene. According to current evidence, mutant DMPK mRNAs containing the trinucleotide expansion are retained in the nucleus, entrapping Muscleblind (MBNL1) protein and several transcription factors in ribonuclear foci and...

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Topics

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Share to DMPK gene
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 21259315
DOI: 10.1002/jnr.22551

Genes

DMPK
CELF1
MBNL1

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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