Supplementary Materials
The PDF file includes:
- Materials and Methods
- Additional authors note
- Fig. S1. Loss of DJ-1 protein in homozygous c.192G>C mutation carriers.
- Fig. S2. Characterization of iPSC and smNPC.
- Fig. S3. Generation of gene-corrected fibroblasts.
- Fig. S4. Schematic of genetic intervention to rescue aberrant splicing of c.192G>C PARK7.
- Fig. S5. Transduction and differentiation of smNPC.
- Fig. S6. Loading controls from Fig. 5 and RECTAS single treatment.
- Fig. S7. Determination of cutoffs for wild_score and maxentscan_change.
- Fig. S8. Western blots.
- Table S1. Features of midbrain organoids extracted from image analysis.
- Table S2. List of primers used for determination of gene expression by CYBR green qPCR.
- Table S3. Hydrolysis probes and primers.
- Legends for data files S1 to S3
- References (46–79)
Other Supplementary Material for this manuscript includes the following:
- Data file S1 (Microsoft Excel format). High-confidence sequence variants in the proband as determined by resequencing of the PARK7 gene.
- Data file S2 (Microsoft Excel format). List of brain-expressed genes harboring variants uniquely identified in cases from both cohorts.
- Data file S3 (Microsoft Excel format). Raw data.
