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 WilliamsLogan M GlasstetterTiffany T JongAbhijeet KapoorSha ZhuYanping ZhuAlexandra GehrleinDavid J VocadloRavi JagasiaJuan J MaruganEllen SidranskyMark J Henderson Yu Chen

Published in: bioRxiv : the preprint server for biology (2024)

pathway, and produced a set of promising potential therapeutics.

Keyphrases

small molecule
protein protein
high throughput
high resolution
risk assessment
replacement therapy
human health