Project highlights: the group identified new non-aminosugar series of glucocerebrosidase inhibitors having chaperone capacity, as well as a probe that is able to inhibit the hydrolytic activity of the N370S mutant form of glucocerebrosidase. During this period, the NCGC has fostered and maintained over 180 active collaborations with both NIH and extramural investigators, facilitating drug discovery efforts across the entire spectrum of human disease. These efforts have led to over 100 high-throughput screens and nearly 60 medicinal chemistry campaigns, providing our collaborators and the general research community a wealth of publications and promising small molecule leads. In addition, the NCGC has undertaken a number of informatic challenges to make better use of existing drug and disease target information and provide the general public with easily accessible resources, further catalyzing the development of new therapies for human disease.
| Mazzulli, Joseph R; Zunke, Friederike; Tsunemi, Taiji et al. (2016) Activation of ?-Glucocerebrosidase Reduces Pathological ?-Synuclein and Restores Lysosomal Function in Parkinson's Patient Midbrain Neurons. J Neurosci 36:7693-706 |
| Aflaki, Elma; Borger, Daniel K; Moaven, Nima et al. (2016) A New Glucocerebrosidase Chaperone Reduces ?-Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism. J Neurosci 36:7441-52 |
| Zhu, Dongqing; Wu, Stephen; Carterette, Ben et al. (2014) Using large clinical corpora for query expansion in text-based cohort identification. J Biomed Inform 49:275-81 |
| Aflaki, Elma; Stubblefield, Barbara K; Maniwang, Emerson et al. (2014) Macrophage models of Gaucher disease for evaluating disease pathogenesis and candidate drugs. Sci Transl Med 6:240ra73 |
| Wieland, Mark L; Wu, Stephen T; Kaggal, Vinod C et al. (2013) Tracking health disparities through natural-language processing. Am J Public Health 103:448-9 |
