Journal article icon

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...

Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

Actions


Access Document


Files:
Publisher copy:
10.1126/scitranslmed.aad9823

Authors


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Pharmacology
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Pharmacology
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Pharmacology
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Pharmacology
Role:
Author
Expand authors...
More from this funder
Funding agency for:
Kirkegaard, T
More from this funder
Funding agency for:
Gray, J
Grant:
105687/Z/14/Z
More from this funder
Funding agency for:
Gray, J
Grant:
105687/Z/14/Z
More from this funder
Funding agency for:
Priestman, D
More from this funder
Funding agency for:
Wallom, K
Expand funders...
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:
2016-09-01
Acceptance date:
2016-08-18
DOI:
EISSN:
1946-6242
ISSN:
1946-6234
Pmid:
27605553
Source identifiers:
641588
Language:
English
Keywords:
Pubs id:
pubs:641588
UUID:
uuid:f8e14c2a-e250-4205-900d-0599eb3c7772
Local pid:
pubs:641588
Deposit date:
2016-10-05

Terms of use


Views and Downloads






If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP