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Article

Validation and Detection of Exon Skipping Boosters in DMD Patient Cell Models and mdx Mouse.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2018

Barthelemy Florian, Wang Dereck, Nelson Stanley F, Miceli M Carrie

Abstract excerpt

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene. Most deletions, duplications, or indels lead to shift of mRNA reading frame, which prevent the production of dystrophin protein. DMD is the leading fatal genetic disorder in childhood. One therapeutic strategy aims to skip one or more exons to restore reading frame to enable the production of internally truncated proteins with partial...

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Share to Cellular Reprogramming
Share to Dystrophin
Share to Genetic Therapy

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Mice, Inbred mdx

Share to Muscle Cells

Share to Muscle Fibers, Skeletal

Share to Muscular Dystrophy, Duchenne

Share to MyoD Protein

Source

Identifiers

PMID: 30171550
DOI: 10.1007/978-1-4939-8651-4_19

Genes

DMD

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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