Research ArticleGene Therapy

Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy

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Science Translational Medicine  22 Jan 2014:
Vol. 6, Issue 220, pp. 220ra10
DOI: 10.1126/scitranslmed.3007523

Article Information

vol. 6 no. 220 220ra10

PubMed: 
Print ISSN: 
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History: 
  • Received for publication September 5, 2013
  • Accepted for publication January 2, 2014

Author Information

  1. Martin K. Childers1,2,*,
  2. Romain Joubert3,
  3. Karine Poulard3,
  4. Christelle Moal3,
  5. Robert W. Grange4,
  6. Jonathan A. Doering4,
  7. Michael W. Lawlor5,6,
  8. Branden E. Rider5,
  9. Thibaud Jamet3,
  10. Nathalie Danièle3,
  11. Samia Martin3,
  12. Christel Rivière3,
  13. Thomas Soker6,
  14. Caroline Hammer3,
  15. Laetitia Van Wittenberghe3,
  16. Mandy Lockard7,
  17. Xuan Guan7,
  18. Melissa Goddard7,
  19. Erin Mitchell7,
  20. Jane Barber7,
  21. J. Koudy Williams7,
  22. David L. Mack1,
  23. Mark E. Furth8,
  24. Alban Vignaud3,
  25. Carole Masurier3,
  26. Fulvio Mavilio3,
  27. Philippe Moullier3,9,10,
  28. Alan H. Beggs5,* and
  29. Anna Buj-Bello3,*
  1. 1Department of Rehabilitation Medicine, School of Medicine, University of Washington, Campus Box 358056, Seattle, WA 98109, USA.
  2. 2Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA.
  3. 3Généthon, 1 bis rue de l’Internationale, 91002 Evry, France.
  4. 4Department of Human Nutrition, Foods and Exercise, Virginia Polytechnic Institute and State University, Blacksburg, VA 24061, USA.
  5. 5Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children’s Hospital, Harvard Medical School, 300 Longwood Avenue, Boston, MA 02115, USA.
  6. 6Department of Pathology and Laboratory Medicine, Children’s Hospital and Medical College of Wisconsin, Milwaukee, WI 53226, USA.
  7. 7Wake Forest Institute for Regenerative Medicine, 391 Technology Way, Winston-Salem, NC 27101, USA.
  8. 8Comprehensive Cancer Center, Wake Forest University Health Sciences, School of Medicine, Medical Center Boulevard, Winston-Salem, NC 27157, USA.
  9. 9INSERM U649, Atlantic Gene Therapies, CHU Hôtel Dieu, 44300 Nantes, France.
  10. 10Molecular Genetics and Microbiology Department, University of Florida, Gainesville, FL 32611, USA.
  1. *Corresponding author. E-mail: abujbello{at}genethon.fr (A.B.-B.); beggs{at}enders.tch.harvard.edu (A.H.B.); mkc8{at}uw.edu (M.K.C.)

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