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Article

Hammerhead ribozyme-mediated destruction of nuclear foci in myotonic dystrophy myoblasts.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 May 2003

Langlois Marc-André, Lee Nan Sook, Rossi John J, Puymirat Jack

Abstract excerpt

Myotonic dystrophy type 1 (DM1) is caused by an unstable CTG expansion in the 3' untranslated region (3'UTR) of the myotonic dystrophy protein kinase gene (DMPK). Transcripts from this altered gene harbor large CUG expansions that are retained in the nucleus of DM1 cells and form foci. It is believed that the formation of these foci is closely linked to DM1 muscle pathogenesis. Here we investigated the...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 12718910
DOI: 10.1016/s1525-0016(03)00068-6

Genes

DMPK

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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Hammerhead ribozyme-mediated destruction of nuclear foci in myotonic dystrophy myoblasts.PMID 12718910Read ↗

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