A Biomarker Study in Patients with GBA1‐Parkinson's Disease and Healthy Controls

• Published in: Movement disorders Vol. 38; no. 5; pp. 783 - 795
• Main Authors: Heijer, Jonas M., Cullen, Valerie C., Pereira, Diana R., Yavuz, Yalcin, Kam, Marieke L., Grievink, Hendrika W., Moerland, Matthijs, Leymarie, Nancy, Khatri, Kshitij, Sollomoni, Imelda, Spitalny, Leslie, Dungeon, Lindsay, Hilt, Dana C., Justman, Craig, Lansbury, Peter, Groeneveld, Geert Jan
• Format: Journal Article
• Language: English
• Published: Hoboken, USA John Wiley & Sons, Inc 01.05.2023; Wiley Subscription Services, Inc
• Subjects: Antigens, CD; Biomarkers; Cerebrospinal fluid; clinical trial; Clinical trials; disease modification; genetic risk factor; glucocerebrosidase; Glucosylceramidase; Glucosylceramidase - genetics; Glucosylceramidase - metabolism; glucosylceramide; Glucosylceramides; glucosylsphingosine; glycosphingolipid; Glycosphingolipids; Humans; Intracellular levels; Isoforms; lactosylceramide; Lactosylceramides; Leukocytes; Leukocytes (granulocytic); Leukocytes (mononuclear); Leukocytes, Mononuclear - metabolism; Lymphocytes; lysosome; Monocytes; Movement disorders; Mutation; neurodegeneration; Neurodegenerative diseases; Parkinson Disease - genetics; Parkinson's disease; Peripheral blood mononuclear cells; Plasma; Principal components analysis; clinical trial; glucosylceramide; neurodegeneration; glycosphingolipid; glucosylsphingosine; lysosome; genetic risk factor; glucocerebrosidase; lactosylceramide; disease modification

Background Molecules related to glucocerebrosidase (GCase) are potential biomarkers for development of compounds targeting GBA1‐associated Parkinson's disease (GBA‐PD). Objectives Assessing variability of various glycosphingolipids (GSLs) in plasma, peripheral blood mononuclear cells (PBMCs), a...