Cited by 173Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusBiomedical Research and Pathophysiology

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

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Kirkegaard, Thomas, Gray, James, Priestman, David A., Wallom, Kerri-Lee, Atkins, Jennifer, Olsen, Ole Dines, Klein, Alexander, Drndarski, Svetlana +12 more·2016·Science Translational Medicine

Abstract

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

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Crossref · 10.1126/scitranslmed.aad9823

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118 of 118 references verified0 citation errors · 0 not checkable

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  • Recombinant HSP70 or arimiclomol reverts lysosomal abnormalities in cells and murine models of various lysosomal storage disorders.

    “In line with this notion, the administration of recombinant HSP70 or the HSP70-inducing agent arimiclomol reverts lysosomal abnormalities in cellula as well as in murine models of various lysosomal storage disorders [789, 791].”

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Research works printed but not linked: 2

  1. [82]Muchowski PJ, Wacker JL. Modulation of neurodegeneration by molecular chaperones. Nat Rev Neurosci. 2005;6:11–22. doi: 10.1038/nrn1587. [DOI] [PubMed] [Google Scholar] · DOI
  2. [86]Ingemann L, Kirkegaard T. Lysosomal storage diseases and the heat shock response: convergences and therapeutic opportunities. J Lipid Res. 2014;55:2198–210. doi: 10.1194/jlr.R048090. [DOI] [PMC free article] [PubMed] [Google Scholar] · DOI

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Authors
Kirkegaard, T., Gray, J., Priestman, D. A., Wallom, K.-L., Atkins, J., Olsen, O. D., Klein, A., Drndarski, S., Petersen, N. H. T., Ingemann, L., Smith, D. A., Morris, L., Bornæs, C., Jørgensen, S. H., Williams, I., Hinsby, A., Arenz, C., Begley, D., Jäättelä, M., & Platt, F. M.
Affiliations
  • Orphazyme (Denmark) (DK)
  • University of Oxford (GB)
  • Humboldt-Universität zu Berlin (DE)
  • King's College London (GB)
  • Danish Cancer Society (DK)
Corresponding author
  • Kirkegaard, Thomas
  • Gray, James
  • Platt, Frances M.
Venue
Science Translational Medicine
Published
2016-09-07
Volume
8
Issue
355
Pages
355ra118-355ra118
ISSN
  • 1946-6234 (ISSN-L)
  • 1946-6242
OpenAlex
W2513570588
Cited by
173
References
118
Citations by year
2026: 9 · 2025: 12 · 2024: 12 · 2023: 27 · 2022: 12 · 2021: 15 · 2020: 22 · 2019: 22 · 2018: 24 · 2017: 11 · 2016: 4 · 2015: 1 · 2014: 1
FWCI
16.35
Type
journal article
Language
en
Open access
Locations
  • Science Translational Medicine (publishedVersion)
  • PubMed (publishedVersion)
  • Europe PMC (PubMed Central) (acceptedVersion)
  • PubMed Central (acceptedVersion)
  • Research Portal (King's College London) (submittedVersion)
Funders
  • Novo Nordisk Foundation
  • Royal Society Wolfson Research
  • Orphazyme
  • Danish Cancer Society (R90-A5783)
  • Danish Medical Research Council (10-083790)
  • European Research Council (AdG 340751)
  • Danish National Research Foundation (DNRF125)
  • Deutsche Forschungsgemeinschaft
  • Wellcome Trust (105687/Z/14/Z)
Topics
  • Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
  • Health Sciences › Medicine › Rheumatology › Glycogen Storage Diseases and Myoclonus
  • Health Sciences › Medicine › Pathology and Forensic Medicine › Biomedical Research and Pathophysiology
Keywords
HSP70, lysosomal storage disease, sphingolipidosis, arimoclomol, glycosphingolipid accumulation, Fabry disease, Sandhoff disease, Niemann-Pick disease type C, neurological symptoms
MeSH terms
Niemann-Pick C1 Protein, Fabry Disease, Animals, Blood-Brain Barrier, Disease Models, Animal, Fibroblasts, Glycosphingolipids, Heat-Shock Proteins, Humans, Hydroxylamines, Injections, Intraperitoneal, Lysosomes, Proteins, Recombinant Proteins, Sphingolipidoses, Tissue Distribution, Disease Progression, Bone Morphogenetic Proteins, Intracellular Signaling Peptides and Proteins, Niemann-Pick Disease, Type C, Administration, Intravenous

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