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Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses
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Increasing Hsp70 expression in lysosomes using the small-molecule arimoclomol ameliorates pathology in several animal models of sphingolipidoses.
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“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].”
From Molecular mechanisms of cell death: recommendations of the nomenclature committee on cell death 2018. (2018) · cites this paper as [791]
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Galluzzi, L., Vitale, I., Aaronson, S. A., Abrams, J. M., Adam, D., Agostinis, P., Alnemri, E. S., Altucci, L., Amelio, I., Andrews, D. W., et al. (2018) · Cell Death & Differentiation · doi:10.1038/s41418-017-0012-4 · Cited by 6,680
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Lysosomal membrane permeabilization and cell death
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Metadata
- 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.
- ORCID
- Venue
- Science Translational Medicine
- Published
- 2016-09-07
- Volume
- 8
- Issue
- 355
- Pages
- 355ra118-355ra118
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- PMC
- PMC6821533
- 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
- FWCI
- 16.35
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- 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 › Genetics (clinical) › Lysosomal Storage Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Biochemistry › Glycogen Storage Diseases and Myoclonus
- Health Sciences › Medicine › Nephrology › 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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
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