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Article

Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.

Stem cell reports - 13 Jan 2015

Li Hongmei Lisa, Fujimoto Naoko, Sasakawa Noriko, Shirai Saya, Ohkame Tokiko, Sakuma Tetsushi, Tanaka Michihiro, Amano Naoki, Watanabe Akira, Sakurai Hidetoshi, Yamamoto Takashi, Yamanaka Shinya, Hotta Akitsu

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe muscle-degenerative disease caused by a mutation in the dystrophin gene. Genetic correction of patient-derived induced pluripotent stem cells (iPSCs) by TALENs or CRISPR-Cas9 holds promise for DMD gene therapy; however, the safety of such nuclease treatment must be determined. Using a unique k-mer database, we systematically identified a unique target region that...

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Identifiers

PMID: 25434822
DOI: 10.1016/j.stemcr.2014.10.013
PMCID: PMC4297888

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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