The DOI matches its Crossref record; 118 of the 118 references it cites were found in a registry or an index.
Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses
Open accessAbstract
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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Cites
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The heat shock protein 70 family: Highly homologous proteins with overlapping and distinct functions
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Self-citationModulation of neurodegeneration by molecular chaperones
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CD91 Is a Common Receptor for Heat Shock Proteins gp96, hsp90, hsp70, and Calreticulin
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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
Self-citationPlatt, F. M., d’Azzo, A., Davidson, B. L., Neufeld, E. F., & Tifft, C. J. (2018) · Nature Reviews Disease Primers · doi:10.1038/s41572-018-0025-4 · Cited by 1,052
Self-citationNeufeld, E. (1991) · Annual Review of Biochemistry · doi:10.1146/annurev.bi.60.070191.001353 · Cited by 924
Lysosomal membrane permeabilization and cell death
Wang, F., Gómez‐Sintes, R., & Boya, P. (2018) · Traffic · doi:10.1111/tra.12613 · Cited by 804
Gu, Y., Liu, Y., Fu, L., Zhai, L., Zhu, J., Han, Y., Jiang, Y., Zhang, Y., Zhang, P., Jiang, Z., et al. (2019) · Nature Medicine · doi:10.1038/s41591-018-0309-y · Cited by 264
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
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