Fig. 4: Relative lysosomal GCase activity in iPSC-derived forebrain neurons.
From: Direct and indirect regulation of β-glucocerebrosidase by the transcription factors USF2 and ONECUT2
Relative lysosomal GCase activity assessed with LysoFQ-GBA1 in live neurons derived from a healthy individual (WT neurons), from a Parkinson patient carrying a heterozygous GBA1 mutation (PDN370S/WT) or a Gaucher patient with compound heterozygous GBA1 mutations (GDL444P/P415R) after overexpression of candidate TFs at 7 (top row) or 14 days (bottom row) post-transduction. LN-229WT (WT), LN-229L444P (L444P), and GBA1-ablated (KO) LN-229 cells. 30 wells/genotype, two independent experiments. AT3375: GCase-selective inhibitor. Six replicate wells/candidate and 26 replicates for the empty vector; N = 1 experiment. Lines: medians and 25-75% quartiles. 100% Lysosomal GCase activity represents the relative activity seen in each of the three cell lines transduced with empty vector. Unpaired t-test.
