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

Thomas Kirkegaard; James Gray; David A. Priestman; Kerri-Lee Wallom; Jennifer Atkins; Ole Dines Olsen; Alexander Klein; Svetlana Drndarski; Nikolaj H. T. Petersen; Linda Ingemann; David A. Smith; Lauren Morris; Claus Bornæs; Signe Humle Jørgensen; Ian Williams; Anders Hinsby; Christoph Arenz; David Begley; Marja Jäättelä; Frances M. Platt

doi:10.1126/scitranslmed.aad9823

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

Thomas Kirkegaard

Orphazyme ApS, Copenhagen, Denmark.

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For correspondence: tkj@orphazyme.com

James Gray

Department of Pharmacology, University of Oxford, Oxford, U.K.

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David A. Priestman

Department of Pharmacology, University of Oxford, Oxford, U.K.

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Kerri-Lee Wallom

Department of Pharmacology, University of Oxford, Oxford, U.K.

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Jennifer Atkins

Department of Pharmacology, University of Oxford, Oxford, U.K.

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Ole Dines Olsen

Orphazyme ApS, Copenhagen, Denmark.Cell Death and Metabolism Unit, Center for Autophagy, Recycling, and Metabolism, Danish Cancer Society Research Center, Copenhagen, Denmark.

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Alexander Klein

Institut für Chemie der Humboldt-Universität zu Berlin, Berlin, Germany.

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Svetlana Drndarski

Institute of Pharmaceutical Science, King’s College London, London, U.K.

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Nikolaj H. T. Petersen

Orphazyme ApS, Copenhagen, Denmark.

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Linda Ingemann

Orphazyme ApS, Copenhagen, Denmark.

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David A. Smith

Department of Pharmacology, University of Oxford, Oxford, U.K.

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Lauren Morris

Department of Pharmacology, University of Oxford, Oxford, U.K.

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Claus Bornæs

Orphazyme ApS, Copenhagen, Denmark.

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Signe Humle Jørgensen

Orphazyme ApS, Copenhagen, Denmark.

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Ian Williams

Department of Pharmacology, University of Oxford, Oxford, U.K.

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Anders Hinsby

Orphazyme ApS, Copenhagen, Denmark.

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Christoph Arenz

Institut für Chemie der Humboldt-Universität zu Berlin, Berlin, Germany.

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David Begley

Institute of Pharmaceutical Science, King’s College London, London, U.K.

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Marja Jäättelä

Cell Death and Metabolism Unit, Center for Autophagy, Recycling, and Metabolism, Danish Cancer Society Research Center, Copenhagen, Denmark.

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Frances M. Platt

Department of Pharmacology, University of Oxford, Oxford, U.K.

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