Pharmacology & ToxicologyProceedings of the National Academy of SciencesPeer-reviewed
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…·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.
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