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Showing 1–2 of 2 results
Advanced filters: Author: Takis Athanasopoulos Clear advanced filters

  • The splicing of the penultimate exon of the dystrophin gene is developmentally regulated. Here the authors show that the dysregulation of this exon’s splicing leads to the expression of an embryonic dystrophin form with a C-terminus distinct from the adult isoform, which leads to muscle wasting in zebrafish and mice.

    • Frédérique Rau
    • Jeanne Lainé
    • Denis Furling
    ResearchOpen Access
    Nature Communications
    Volume: 6, P: 1-10

  • Duchenne muscular dystrophy is a progressive degenerative disease of muscles caused by mutations in the dystrophin gene. Here the authors use AAV vectors to deliver microdystrophin to dogs with muscular dystrophy, and show restoration of dystrophin expression and reduction of symptoms up to 26 months of age.

    • Caroline Le Guiner
    • Laurent Servais
    • George Dickson
    ResearchOpen Access
    Nature Communications
    Volume: 8, P: 1-15