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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Author

Listed:
  • Thurman M. Wheeler

    (University of Rochester, 601 Elmwood Avenue, Rochester, New York, 14642, USA
    Center for Neural Development and Disease, University of Rochester, 601 Elmwood Avenue, Rochester, New York, 14642, USA)

  • Andrew J. Leger

    (Genzyme Corporation, 49 New York Avenue)

  • Sanjay K. Pandey

    (Isis Pharmaceuticals, 2855 Gazelle Court)

  • A. Robert MacLeod

    (Isis Pharmaceuticals, 2855 Gazelle Court)

  • Masayuki Nakamori

    (University of Rochester, 601 Elmwood Avenue, Rochester, New York, 14642, USA
    Center for Neural Development and Disease, University of Rochester, 601 Elmwood Avenue, Rochester, New York, 14642, USA)

  • Seng H. Cheng

    (Genzyme Corporation, 49 New York Avenue)

  • Bruce M. Wentworth

    (Genzyme Corporation, 49 New York Avenue)

  • C. Frank Bennett

    (Isis Pharmaceuticals, 2855 Gazelle Court)

  • Charles A. Thornton

    (University of Rochester, 601 Elmwood Avenue, Rochester, New York, 14642, USA
    Center for Neural Development and Disease, University of Rochester, 601 Elmwood Avenue, Rochester, New York, 14642, USA)

Abstract

Nuclear-retained transcripts containing expanded repeats are shown to be sensitive to antisense silencing, and in a transgenic mouse model of myotonic dystrophy type 1, systemic administration of ASOs causes a rapid knockdown of the toxic RNA in skeletal muscle, correcting some hallmark features of the disease.

Suggested Citation

  • Thurman M. Wheeler & Andrew J. Leger & Sanjay K. Pandey & A. Robert MacLeod & Masayuki Nakamori & Seng H. Cheng & Bruce M. Wentworth & C. Frank Bennett & Charles A. Thornton, 2012. "Targeting nuclear RNA for in vivo correction of myotonic dystrophy," Nature, Nature, vol. 488(7409), pages 111-115, August.
    • Handle: RePEc:nat:nature:v:488:y:2012:i:7409:d:10.1038_nature11362
    DOI: 10.1038/nature11362
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    Cited by:

    1. Ningyan Hu & Eunjoo Kim & Layal Antoury & Thurman M. Wheeler, 2023. "Correction of Clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy," Nature Communications, Nature, vol. 14(1), pages 1-15, December.

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