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Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in.

Human gene therapy - 1 Jul 2013

Popplewell Linda, Koo Taeyoung, Leclerc Xavier, Duclert Aymeric, Mamchaoui Kamel, Gouble Agnés, Mouly Vincent, Voit Thomas, Pâques Frédéric, Cédrone Frédéric, Isman Olga, Yáñez-Muñoz Rafael J, Dickson George

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe inherited, muscle-wasting disorder caused by mutations in the DMD gene. Gene therapy development for DMD has concentrated on vector-based DMD minigene transfer, cell-based gene therapy using genetically modified adult muscle stem cells or healthy wild-type donor cells, and antisense oligonucleotide-induced exon-skipping therapy to restore the reading frame of the...

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Share to Deoxyribonucleases
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 23790397
DOI: 10.1089/hum.2013.081

Genes

DMD

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