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Article

Restoration of Dystrophin Protein Expression by Exon Skipping Utilizing CRISPR-Cas9 in Myoblasts Derived from DMD Patient iPS Cells.

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

Ifuku Masataka, Iwabuchi Kumiko A, Tanaka Masami, Lung Mandy Siu Yu, Hotta Akitsu

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a congenital X-linked disease caused by mutations in the gene encoding the dystrophin protein, which is required for myofiber integrity. Exon skipping therapy is an emerging strategy for restoring the open reading frame of the dystrophin gene to produce functional protein in DMD patients by skipping single or multiple exons. Although antisense oligonucleotides are able to...

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Source

Identifiers

PMID: 30171543
DOI: 10.1007/978-1-4939-8651-4_12

Genes

DMD

Provenance

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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