Mechanistic and Biochemical Studies of Glucocerebrosidase
Mutations in the enzyme glucocerebrosidase (GBA1) are the most common genetic risk factor for development of Parkinson’s disease (PD). PD is characterized by the buildup of abnormal protein deposits in the brain, followed by progressive loss of neurons and behavioural symptoms. Numerous studies have noted a correlation between reduced GBA1 activity and increased levels of these abnormal protein deposits in the brain, but the relationship remains poorly understood. The aim of this project is to investigate whether augmenting levels of GBA1 can prevent the accumulation of abnormal proteins in the brain. The success of this project may lead to a better understanding of the causes of PD and could ultimately result in new strategies to develop disease-modifying therapies for Parkinson’s disease.