Research ArticleNeurodegenerative Disease

Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

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Science Translational Medicine  25 Feb 2015:
Vol. 7, Issue 276, pp. 276ra26
DOI: 10.1126/scitranslmed.3010101

Article Information

vol. 7 no. 276 276ra26

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication July 21, 2014
  • Accepted for publication February 5, 2015
  • .

Author Information

  1. Charles H. Vite1,*,
  2. Jessica H. Bagel1,
  3. Gary P. Swain1,
  4. Maria Prociuk1,
  5. Tracey U. Sikora2,
  6. Veronika M. Stein1,,
  7. Patricia O’Donnell2,
  8. Therese Ruane2,
  9. Sarah Ward1,
  10. Alexandra Crooks1,
  11. Su Li1,,
  12. Elizabeth Mauldin2,
  13. Susan Stellar3,
  14. Marc De Meulder4,
  15. Mark L. Kao3,
  16. Daniel S. Ory5,
  17. Cristin Davidson6,
  18. Marie T. Vanier7 and
  19. Steven U. Walkley6
  1. 1Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  2. 2Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
  3. 3Janssen Research & Development, LLC, Janssen Pharmaceutical Companies of Johnson and Johnson, Titusville, NJ 08560, USA.
  4. 4Janssen Research & Development, a division of Janssen Pharmaceutica NV, Janssen Pharmaceutical Companies of Johnson and Johnson, Beerse, Belgium.
  5. 5Diabetic Cardiovascular Disease Center, Washington University School of Medicine, St. Louis, MO 63110, USA.
  6. 6Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Intellectual and Developmental Disabilities Research Center, Albert Einstein College of Medicine, Bronx, NY 10461, USA.
  7. 7INSERM U820; EA4611, Université Claude Bernard Lyon 1, Lyon, France.
  1. *Corresponding author. E-mail: vite{at}vet.upenn.edu
  • Present address: Department of Small Animal Medicine and Surgery, University of Veterinary Medicine Hannover, Niedersachsen, Germany.

  • Present address: College of Veterinary Medicine, Kansas State University, Manhattan, KS 66506, USA.

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