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Research ArticleLysosomal Storage Diseases
Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses
- Thomas Kirkegaard1,*,†,
- James Gray2,*,
- David A. Priestman2,
- Kerri-Lee Wallom2,
- Jennifer Atkins2,
- Ole Dines Olsen1,3,
- Alexander Klein4,
- Svetlana Drndarski5,
- Nikolaj H. T. Petersen1,
- Linda Ingemann1,
- David A. Smith2,
- Lauren Morris2,
- Claus Bornæs1,
- Signe Humle Jørgensen1,
- Ian Williams2,
- Anders Hinsby1,
- Christoph Arenz4,
- David Begley5,
- Marja Jäättelä3,* and
- Frances M. Platt2,*
- 1Orphazyme ApS, Copenhagen, Denmark.
- 2Department of Pharmacology, University of Oxford, Oxford, U.K.
- 3Cell Death and Metabolism Unit, Center for Autophagy, Recycling, and Metabolism, Danish Cancer Society Research Center, Copenhagen, Denmark.
- 4Institut für Chemie der Humboldt-Universität zu Berlin, Berlin, Germany.
- 5Institute of Pharmaceutical Science, King’s College London, London, U.K.
- ↵†Corresponding author. Email: tkj{at}orphazyme.com
↵* These authors contributed equally to this work.
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Science Translational Medicine 07 Sep 2016:
Vol. 8, Issue 355, pp. 355ra118
DOI: 10.1126/scitranslmed.aad9823
Vol. 8, Issue 355, pp. 355ra118
DOI: 10.1126/scitranslmed.aad9823
Thomas Kirkegaard
1Orphazyme ApS, Copenhagen, Denmark.
James Gray
2Department of Pharmacology, University of Oxford, Oxford, U.K.
David A. Priestman
2Department of Pharmacology, University of Oxford, Oxford, U.K.
Kerri-Lee Wallom
2Department of Pharmacology, University of Oxford, Oxford, U.K.
Jennifer Atkins
2Department of Pharmacology, University of Oxford, Oxford, U.K.
Ole Dines Olsen
1Orphazyme ApS, Copenhagen, Denmark.
3Cell Death and Metabolism Unit, Center for Autophagy, Recycling, and Metabolism, Danish Cancer Society Research Center, Copenhagen, Denmark.
Alexander Klein
4Institut für Chemie der Humboldt-Universität zu Berlin, Berlin, Germany.
Svetlana Drndarski
5Institute of Pharmaceutical Science, King’s College London, London, U.K.
Nikolaj H. T. Petersen
1Orphazyme ApS, Copenhagen, Denmark.
Linda Ingemann
1Orphazyme ApS, Copenhagen, Denmark.
David A. Smith
2Department of Pharmacology, University of Oxford, Oxford, U.K.
Lauren Morris
2Department of Pharmacology, University of Oxford, Oxford, U.K.
Claus Bornæs
1Orphazyme ApS, Copenhagen, Denmark.
Signe Humle Jørgensen
1Orphazyme ApS, Copenhagen, Denmark.
Ian Williams
2Department of Pharmacology, University of Oxford, Oxford, U.K.
Anders Hinsby
1Orphazyme ApS, Copenhagen, Denmark.
Christoph Arenz
4Institut für Chemie der Humboldt-Universität zu Berlin, Berlin, Germany.
David Begley
5Institute of Pharmaceutical Science, King’s College London, London, U.K.
Marja Jäättelä
3Cell Death and Metabolism Unit, Center for Autophagy, Recycling, and Metabolism, Danish Cancer Society Research Center, Copenhagen, Denmark.
Frances M. Platt
2Department of Pharmacology, University of Oxford, Oxford, U.K.
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