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