Journal
SYNAPSE
Volume 71, Issue 7, Pages -Publisher
WILEY
DOI: 10.1002/syn.21967
Keywords
ambroxol; glucocerebrosidase; Parkinson disease; nonhuman primate
Categories
Funding
- Javon Trust, Medical Research Council (MRC) CoEN [MR/L501499/1]
- MRC Experimental Medicine Program [MR/M006646/1]
- Parkinson UK [G-1403]
- Kattan Trust
- NIHR University College London Hospitals Biomedical Research Centre
- MRC [MR/M006646/1] Funding Source: UKRI
- Medical Research Council [MR/M006646/1, MR/L501499/1] Funding Source: researchfish
- National Institute for Health Research [NF-SI-0611-10237] Funding Source: researchfish
- Parkinson's UK [G-1403] Funding Source: researchfish
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Mutations in the glucocerebrosidase 1 (GBA1) gene are related to both Parkinson disease (PD) and Gaucher disease (GD). In both cases, the condition is associated with deficiency of glucocerebrosidase (GCase), the enzyme encoded by GBA1. Ambroxol is a small molecule chaperone that has been shown in mice to cross the blood-brain barrier, increase GCase activity and reduce alpha-synuclein protein levels. In this study, we analyze the effect of ambroxol treatment on GCase activity in healthy nonhuman primates. We show that daily administration of ambroxol results in increased brain GCase activity. Our work further indicates that ambroxol should be investigated as a novel therapy for both PD and neuronopathic GD in humans.
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