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Increasing Glucocerebrosidase Activity as a Therapeutic Strategy

Mutations in GBA1, the gene encoding the protein glucocerebrosidase, represent a common genetic risk factor for developing Parkinson disease (PD). PD patients with and without GBA1 mutations exhibit lower levels of glucocerebrosidase in the central nervous system (CNS), suggesting a strong correlation between low glucocerebrosidase activity and the development of the disease. Hence, augmenting glucocerebrosidase activity in the CNS represents a plausible strategy for treating PD.

Project Description:
This project will identify small molecule drugs that increase the activity of glucocerebrosidase in the CNS and evaluate the efficacy of these small molecule drugs in disease models.

Relevance to Diagnosis/Treatment of Parkinson’s Disease:                     
Previous research supports the notion that increasing glucocerebrosidase activity can lower alpha-synuclein levels and associated toxicity. Hence, increasing glucocerebrosidase activity in the CNS may slow disease progression.

Anticipated Outcome:          
The successful outcome of these studies should lead to the identification of small molecule drugs that increase CNS glucocerebrosidase activity and thereby decrease alpha-synuclein levels.


  • Sergio Pablo Sardi, PharmD, PhD

    Framingham, MA United States

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