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Article

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

Human mutation - 1 Mar 2009

Aartsma-Rus Annemieke, Fokkema Ivo, Verschuuren Jan, Ginjaar Ieke, van Deutekom Judith, van Ommen Gert-Jan, den Dunnen Johan T

Abstract excerpt

Antisense-mediated exon skipping aiming for reading frame restoration is currently a promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach is mutation specific, but as the majority of DMD patients have deletions that cluster in hotspot regions, the skipping of a small number of exons is applicable to relatively large numbers of patients. To assess the actual applicability of the...

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Source

Identifiers

PMID: 19156838
DOI: 10.1002/humu.20918

Genes

DMD

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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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