CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease

CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease

Molecular Therapy 24,
1019 (June 2016). doi:10.1038/mt.2016.53

Authors: John Marshall, Ying Sun, Dinesh S Bangari, Eva Budman, Hyejung Park, Jennifer B Nietupski, Amy Allaire, Mary A Cromwell, Bing Wang, Gregory A Grabowski, John P Leonard & Seng H Cheng

Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Authors: John MarshallYing SunDinesh S BangariEva BudmanHyejung ParkJennifer B NietupskiAmy AllaireMary A CromwellBing WangGregory A GrabowskiJohn P LeonardSeng H Cheng Source Type: research