THERAPEUTIC CLASS

Ambroxol Effects in Glucocerebrosidase and a-Synuclein Transgenic Mice

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Gaucher disease is caused by mutations in the glucocerebrosidase 1 gene that result in deficiency of the lysosomal enzyme glucocerebrosidase. Both homozygous and heterozygous glucocerebrosidase 1 mutations confer an increased risk for developing Parkinson disease. Current estimates indicate that 10 to 25% of Parkinson patients carry glucocerebrosidase 1 mutations. Ambroxol is a small molecule chaperone that has been shown to increase glucocerebrosidase activity in vitro. This study investigated the effect of ambroxol treatment on glucocerebrosidase activity and on a-synuclein and phosphorylated a-synuclein protein levels in mice

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: .,

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Language

English

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Classification

NONE

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Subject(s)

Central Nervous System

Specific Detail Info

ANN NEUROL 2016;80:766–775

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