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Article

Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model.

PloS one - 1 Jan 2020

Yavas Alper, Weij Rudie, van Putten Maaike, Kourkouta Eleni, Beekman Chantal, Puoliväli Jukka, Bragge Timo, Ahtoniemi Toni, Knijnenburg Jeroen, Hoogenboom Marlies Elisabeth, Ariyurek Yavuz, Aartsma-Rus Annemieke, van Deutekom Judith, Datson Nicole

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disorder caused by reading frame disrupting mutations in the DMD gene leading to absence of functional dystrophin. Antisense oligonucleotide (AON)-mediated exon skipping is a therapeutic approach aimed at restoring the reading frame at the pre-mRNA level, allowing the production of internally truncated partly functional dystrophin proteins....

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 33362201
DOI: 10.1371/journal.pone.0244215
PMCID: PMC7757897

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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.

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