Single cell analysis of iPSC-derived midbrain organoids (GBA1 mutant and controls). Single cell analysis of iPSC-derived midbrain organoids (GBA1 mutant and controls)

Mutations in the lysosomal enzyme β-glucocerebrosidase (GCase), which cause Gaucher´s disease, are the most frequent genetic risk factor for Parkinson’s disease (PD). Here, we employed single-cell genomic approaches in induced pluripotent stem cell (iPSC)-derived midbrain organoids to dissect the mechanisms underlying GCase-related neurodegeneration. Overall design: Midbran organoids generated from L444P-GBA1 and one E326K-GBA1 human iPSCs lines and corresponding gene corrected lines. For each condition, we prepared single-cell suspensions with a pool of 4 organoids each, all with similar sizes (Day 40).