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Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

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Science Translational Medicine  02 Mar 2011:
Vol. 3, Issue 72, pp. 72ra18
DOI: 10.1126/scitranslmed.3001777

Article Information

vol. 3 no. 72 72ra18

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication October 6, 2010
  • Accepted for publication February 10, 2011
  • .

Author Information

  1. Marco A. Passini1,*,
  2. Jie Bu1,
  3. Amy M. Richards1,
  4. Cathrine Kinnecom1,
  5. S. Pablo Sardi1,
  6. Lisa M. Stanek1,
  7. Yimin Hua2,
  8. Frank Rigo3,
  9. John Matson3,
  10. Gene Hung3,
  11. Edward M. Kaye1,4,
  12. Lamya S. Shihabuddin1,
  13. Adrian R. Krainer2,
  14. C. Frank Bennett3 and
  15. Seng H. Cheng1
  1. 1Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.
  2. 2Cold Spring Harbor Laboratory, 1 Bungtown Road, Cold Spring Harbor, NY 11724, USA.
  3. 3Isis Pharmaceuticals, 1896 Rutherford Road, Carlsbad, CA 92008, USA.
  4. 4Department of Neurology, Harvard Medical School, 300 Longwood Avenue, Boston, MA 02114, USA.
  1. *To whom correspondence should be addressed. E-mail: marco.passini{at}genzyme.com

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