Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses-Reference-Cited by-同舟云学术

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

Published:2016-09-07 Issue:355 Volume:8 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Kirkegaard Thomas¹,Gray James²,Priestman David A.²,Wallom Kerri-Lee²,Atkins Jennifer²,Olsen Ole Dines¹³,Klein Alexander⁴,Drndarski Svetlana⁵,Petersen Nikolaj H. T.¹,Ingemann Linda¹,Smith David A.²,Morris Lauren²,Bornæs Claus¹,Jørgensen Signe Humle¹,Williams Ian²,Hinsby Anders¹,Arenz Christoph⁴,Begley David⁵,Jäättelä Marja³,Platt Frances M.²

Affiliation:

1. Orphazyme ApS, Copenhagen, Denmark.

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

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

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

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

Abstract

Increasing Hsp70 expression in lysosomes using the small-molecule arimoclomol ameliorates pathology in several animal models of sphingolipidoses.

Funder

Novo Nordisk Foundation

Royal Society Wolfson Research

Orphazyme