Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Thurman M. Wheeler, Andrew J. Leger, Sanjay K. Pandey, A. Robert MacLeod, Masayuki Nakamori, Seng H. Cheng, Bruce M. Wentworth, C. Frank Bennett and Charles A. Thornton ()
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Thurman M. Wheeler: 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
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

Nature, 2012, vol. 488, issue 7409, 111-115

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.

Date: 2012
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DOI: 10.1038/nature11362

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