Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for...
Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders
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Publisher
United States: Elsevier Inc
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Language
English
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Publisher
United States: Elsevier Inc
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Scope and Contents
Contents
Lysosomal storage disorders (LSDs) are a group of inborn metabolic diseases caused by mutations in genes that encode proteins involved in different lysosomal functions, in most instances acidic hydrolases. Different therapeutic approaches have been developed to treat these disorders. Pharmacological chaperone therapy (PCT) is an emerging approach b...
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Full title
Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4817787
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4817787
Other Identifiers
ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1038/mt.2015.62
