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Showing 1–2 of 2 results
Advanced filters: Author: Hichem Tasfaout Clear advanced filters

  • X-linked myotubular myopathy is caused by mutations in the gene coding for myotubularin 1, and is characterized by overexpression of dynamin 2. Here the authors develop antisense oligonucleotides to dynamin 2, and show that systemic injection leads to improved pathology in mice.

    • Hichem Tasfaout
    • Suzie Buono
    • Jocelyn Laporte
    ResearchOpen Access
    Nature Communications
    Volume: 8, P: 1-13

  • A method is developed for expressing large dystrophins to enhance muscle function in mouse models of muscular dystrophy, with potential clinical benefits for numerous disorders caused by mutations in large genes that exceed the adeno-associated virus capacity.

    • Hichem Tasfaout
    • Christine L. Halbert
    • Jeffrey S. Chamberlain
    Research
    Nature
    Volume: 632, P: 192-200