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
D. Williams, L. M. Glasstetter, T. T. Jong, A. Kapoor, S. Zhu, Y. Zhu, A. Gehrlein, D. J. Vocadlo, R. Jagasia, J. J. Marugan, E. Sidransky, M. J. Henderson, Y. 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.
bioRxiv · Mar 22, 2024 · PreprintPharmacology & Toxicology