Research ArticleCystic Fibrosis

In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis

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Science Translational Medicine  27 Mar 2019:
Vol. 11, Issue 485, eaau7531
DOI: 10.1126/scitranslmed.aau7531

Article Information

vol. 11 no. 485

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication July 11, 2018
  • Accepted for publication March 1, 2019
  • .

Author Information

  1. Xingshen Sun1,
  2. Yaling Yi1,
  3. Ziying Yan1,
  4. Bradley H. Rosen1,2,
  5. Bo Liang1,
  6. Michael C. Winter1,
  7. T. Idil Apak Evans1,
  8. Pavana G. Rotti1,
  9. Yu Yang1,
  10. Jaimie S. Gray1,
  11. Soo Yeun Park1,
  12. Weihong Zhou1,
  13. Yulong Zhang1,
  14. Shashanna R. Moll1,
  15. Lisa Woody3,
  16. Dao M. Tran3,
  17. Licong Jiang3,
  18. Annelotte M. Vonk4,
  19. Jeffrey M. Beekman4,
  20. Paul Negulescu3,
  21. Fred Van Goor3,
  22. Dennis F. Fiorino3,
  23. Katherine N. Gibson-Corley5 and
  24. John F. Engelhardt1,2,*
  1. 1Department of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
  2. 2Department of Internal Medicine, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
  3. 3Vertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA.
  4. 4Pediatric Pulmonology and Regenerative Medicine Center, University Medical Center Utrecht, Utrecht, Netherlands.
  5. 5Department of Pathology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
  1. *Corresponding author. Email: john-engelhardt{at}uiowa.edu

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Article usage: March 2019 to September 2019

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May 20192227167
Jun 20192649576
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