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AAV-mediated gene transfer restores a normal muscle transcriptome in a canine model of X-linked myotubular myopathy

Jean-Baptiste Dupont, Jianjun Guo, Michael W. Lawlor, Robert W. Grange, John T. Gray, Ana Buj-Bello, Martin K. Childers, David L. Mack
doi: https://doi.org/10.1101/499384
Jean-Baptiste Dupont
1Departments of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA.
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Jianjun Guo
3Audentes Therapeutics, San Francisco, CA 94108, USA.
7Present address: Clinical NGS Group, Thermo Fisher Scientific, South San Francisco, CA 94080, USA.
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Michael W. Lawlor
4Department of Pathology and Laboratory Medicine and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI 53226, USA.
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Robert W. Grange
5Department of Human Nutrition, Foods, and Exercise, Virginia Polytechnic Institute and State University, Blacksburg, VA 24060, USA.
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John T. Gray
3Audentes Therapeutics, San Francisco, CA 94108, USA.
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Ana Buj-Bello
6Généthon, INSERM UMR S951, Université Evry Val-d’Essone, Université Paris-Saclay, 91000 Evry, France.
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Martin K. Childers
1Departments of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA.
8Present address: AskBio, Chapel Hill, NC 27514, USA.
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David L. Mack
1Departments of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA.
2Department of Bioengineering, University of Washington, Seattle, WA 98109, USA.
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  • For correspondence: dmack21@uw.edu
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Article Information

doi 
https://doi.org/10.1101/499384
History 
  • December 17, 2018.
Copyright 
The copyright holder for this preprint is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. All rights reserved. No reuse allowed without permission.

Author Information

  1. Jean-Baptiste Dupont1,
  2. Jianjun Guo3,7,
  3. Michael W. Lawlor4,
  4. Robert W. Grange5,
  5. John T. Gray3,
  6. Ana Buj-Bello6,
  7. Martin K. Childers1,8 and
  8. David L. Mack1,2,*
  1. 1Departments of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA.
  2. 2Department of Bioengineering, University of Washington, Seattle, WA 98109, USA.
  3. 3Audentes Therapeutics, San Francisco, CA 94108, USA.
  4. 4Department of Pathology and Laboratory Medicine and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI 53226, USA.
  5. 5Department of Human Nutrition, Foods, and Exercise, Virginia Polytechnic Institute and State University, Blacksburg, VA 24060, USA.
  6. 6Généthon, INSERM UMR S951, Université Evry Val-d’Essone, Université Paris-Saclay, 91000 Evry, France.
  7. 7Present address: Clinical NGS Group, Thermo Fisher Scientific, South San Francisco, CA 94080, USA.
  8. 8Present address: AskBio, Chapel Hill, NC 27514, USA.
  1. ↵*Corresponding Author: dmack21{at}uw.edu
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Posted December 17, 2018.
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AAV-mediated gene transfer restores a normal muscle transcriptome in a canine model of X-linked myotubular myopathy
Jean-Baptiste Dupont, Jianjun Guo, Michael W. Lawlor, Robert W. Grange, John T. Gray, Ana Buj-Bello, Martin K. Childers, David L. Mack
bioRxiv 499384; doi: https://doi.org/10.1101/499384
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AAV-mediated gene transfer restores a normal muscle transcriptome in a canine model of X-linked myotubular myopathy
Jean-Baptiste Dupont, Jianjun Guo, Michael W. Lawlor, Robert W. Grange, John T. Gray, Ana Buj-Bello, Martin K. Childers, David L. Mack
bioRxiv 499384; doi: https://doi.org/10.1101/499384

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