Research ArticleLysosomal Storage Diseases

Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses

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Science Translational Medicine  07 Sep 2016:
Vol. 8, Issue 355, pp. 355ra118
DOI: 10.1126/scitranslmed.aad9823

Article Information

vol. 8 no. 355 355ra118

PubMed: 
Print ISSN: 
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History: 
  • Received for publication December 2, 2015
  • Accepted for publication August 18, 2016

Author Information

  1. Thomas Kirkegaard1,*,,
  2. James Gray2,*,
  3. David A. Priestman2,
  4. Kerri-Lee Wallom2,
  5. Jennifer Atkins2,
  6. Ole Dines Olsen1,3,
  7. Alexander Klein4,
  8. Svetlana Drndarski5,
  9. Nikolaj H. T. Petersen1,
  10. Linda Ingemann1,
  11. David A. Smith2,
  12. Lauren Morris2,
  13. Claus Bornæs1,
  14. Signe Humle Jørgensen1,
  15. Ian Williams2,
  16. Anders Hinsby1,
  17. Christoph Arenz4,
  18. David Begley5,
  19. Marja Jäättelä3,* and
  20. Frances M. Platt2,*
  1. 1Orphazyme ApS, Copenhagen, Denmark.
  2. 2Department of Pharmacology, University of Oxford, Oxford, U.K.
  3. 3Cell Death and Metabolism Unit, Center for Autophagy, Recycling, and Metabolism, Danish Cancer Society Research Center, Copenhagen, Denmark.
  4. 4Institut für Chemie der Humboldt-Universität zu Berlin, Berlin, Germany.
  5. 5Institute of Pharmaceutical Science, King’s College London, London, U.K.
  1. Corresponding author. Email: tkj{at}orphazyme.com
    • * These authors contributed equally to this work.

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