Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
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- Marco A. Passini
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
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- Jie Bu
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
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- Amy M. Richards
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
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- Cathrine Kinnecom
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
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- S. Pablo Sardi
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
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- Lisa M. Stanek
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
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- Yimin Hua
- Cold Spring Harbor Laboratory, 1 Bungtown Road, Cold Spring Harbor, NY 11724, USA.
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- Frank Rigo
- Isis Pharmaceuticals, 1896 Rutherford Road, Carlsbad, CA 92008, USA.
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- John Matson
- Isis Pharmaceuticals, 1896 Rutherford Road, Carlsbad, CA 92008, USA.
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- Gene Hung
- Isis Pharmaceuticals, 1896 Rutherford Road, Carlsbad, CA 92008, USA.
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- Edward M. Kaye
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
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- Lamya S. Shihabuddin
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
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- Adrian R. Krainer
- Cold Spring Harbor Laboratory, 1 Bungtown Road, Cold Spring Harbor, NY 11724, USA.
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- C. Frank Bennett
- Isis Pharmaceuticals, 1896 Rutherford Road, Carlsbad, CA 92008, USA.
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- Seng H. Cheng
- Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
書誌事項
- 公開日
- 2011-03-02
- DOI
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- 10.1126/scitranslmed.3001777
- 公開者
- American Association for the Advancement of Science (AAAS)
この論文をさがす
説明
<jats:p>Central nervous system–directed antisense therapy ameliorates symptoms in a severe neuromuscular disorder in mice.</jats:p>
収録刊行物
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- Science Translational Medicine
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Science Translational Medicine 3 (72), 72ra18-, 2011-03-02
American Association for the Advancement of Science (AAAS)
