Journal article
Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.
- Abstract:
-
Lysosomal storage diseases (LSDs) often manifest with severe systemic and central nervous system (CNS) symptoms. The existing treatment options are limited and have no or only modest efficacy against neurological manifestations of disease. We demonstrate that recombinant human heat shock protein 70 (HSP70) improves the binding of several sphingolipid-degrading enzymes to their essential cofactor bis(monoacyl)glycerophosphate in vitro. HSP70 treatment reversed lysosomal pathology in primary fi...
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- Publication status:
- Published
- Peer review status:
- Peer reviewed
- Version:
- Accepted Manuscript
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(pdf, 4.0MB)
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- Publisher copy:
- 10.1126/scitranslmed.aad9823
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Authors
Funding
Bibliographic Details
- Publisher:
- American Association for the Advancement of Science Publisher's website
- Journal:
- Science translational medicine Journal website
- Volume:
- 8
- Issue:
- 355
- Pages:
- 355ra118
- Publication date:
- 2016-09-05
- Acceptance date:
- 2016-08-18
- DOI:
- EISSN:
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1946-6242
- ISSN:
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1946-6234
- Pubs id:
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pubs:641588
- URN:
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uri:f8e14c2a-e250-4205-900d-0599eb3c7772
- UUID:
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uuid:f8e14c2a-e250-4205-900d-0599eb3c7772
- Local pid:
- pubs:641588
Item Description
- Language:
- English
- Keywords:
Terms of use
- Copyright holder:
- Copyright © 2016, American Association for the Advancement of Science
- Copyright date:
- 2016
- Notes:
- Copyright © 2016, American Association for the Advancement of Science. This is the author accepted manuscript following peer review version of the article. The final version is available online from American Association for the Advancement of Science at: 10.1126/scitranslmed.aad9823
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