Development of quantitative high-throughput screening assays to identify, validate, and optimize small-molecule stabilizers of misfolded β-glucocerebrosidase with therapeutic potential for Gaucher disease and Parkinson's disease
Darian Williams, Logan M. Glasstetter, Tiffany T. Jong, Tiffany Chen, Abhijeet Kapoor, Sha Zhu, Yanping Zhu, Raul Calvo, Alexandra Gehrlein, Kimberly Wong, Andrew N. Hogan, David J. Vocadlo, Ravi Jagasia, Juan Marugán, Ellen Sidransky, , Yu Chen
This paper develops high-throughput assays to identify and optimize small-molecule stabilizers of misfolded β-glucocerebrosidase, showing NCGC326 reverses pathological accumulation and synergizes with proteostasis regulators.
Proceedings of the National Academy of Sciences · Oct 10, 2024Peer-reviewedPharmacology & Toxicology