iSCORE-PD: an isogenic stem cell collection to research Parkinson's disease - PubMed
3 hours ago
- #genome editing
- #Parkinson's disease
- #stem cells
- iSCORE-PD is a collection of 65 genome-edited human pluripotent stem cell (hPSC) lines designed to study Parkinson's disease (PD).
- All lines are derived from a well-characterized female human embryonic stem cell line and carry disease-causing or high-risk variants in 11 PD-linked genes.
- The collection undergoes extensive quality control, with whole-genome sequencing showing minimal genetic variation between lines compared to patient-derived iPSCs.
- Random mutations from cell culture, not genome-editing off-target effects, are the main source of variation, and using multiple independent clones per mutation can control for this drift.
- The resource highlights the advantages of prime editing over conventional CRISPR/Cas9 methods and establishes best practices for generating disease-modeling hPSC collections.
- iSCORE-PD is publicly available and funded by grants from the National Institutes of Health (NIH).