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Chemical chaperone therapy: clinical effect in murine G M1 ‐gangliosidosis
Ist Teil von
Annals of neurology, 2007-12, Vol.62 (6), p.671-675
Erscheinungsjahr
2007
Quelle
Wiley Online Library All Journals
Beschreibungen/Notizen
Abstract
Certain low‐molecular‐weight substrate analogs act both as in vitro competitive inhibitors of lysosomal hydrolases and as intracellular enhancers (chemical chaperones) by stabilization of mutant proteins. In this study, we performed oral administration of a chaperone compound
N
‐octyl‐4‐epi‐β‐valienamine to G
M1
‐gangliosidosis model mice expressing R201C mutant human β‐galactosidase. A newly developed neurological scoring system was used for clinical assessment.
N
‐Octyl‐4‐epi‐β‐valienamine was delivered rapidly to the brain, increased β‐galactosidase activity, decreased ganglioside G
M1
, and prevented neurological deterioration within a few months. No adverse effect was observed during this experiment.
N
‐Octyl‐4‐epi‐β‐valienamine will be useful for chemical chaperone therapy of human G
M1
‐gangliosidosis. Ann Neurol 2007
Sprache
Englisch
Identifikatoren
ISSN: 0364-5134
eISSN: 1531-8249
DOI: 10.1002/ana.21284
Titel-ID: cdi_crossref_primary_10_1002_ana_21284
Format
–
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