Image of Ambroxol Effects in Glucocerebrosidase and a-Synuclein Transgenic Mice

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Ambroxol Effects in Glucocerebrosidase and a-Synuclein Transgenic Mice


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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REQJ662-01Titan CenterAvailable

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English
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NONE
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ANN NEUROL 2016;80:766–775
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