Modulators of GCase
A genome-wide CRISPR-activation screen in GBA L444P cells identifies modifiers of β-glucocerebrosidase (GCase), a central pathway in Parkinson’s disease.
Validation Pipeline
Validation uses iPSC-derived neurons, LysoFQ-GBA live assays, lipidomics (GlcCer/GlcSph), and α-synuclein seeding assays to find druggable nodes.