BiophysicsbioRxiv
Protein structural features predict responsiveness to pharmacological chaperone treatment for three lysosomal storage disorders
J. C. Woodard, W. Zheng, Y. Zhang
The paper uses structural and sequence data to predict responsiveness to pharmacological chaperone treatment for Gaucher disease variants, finding that protein topology and mutation stability influence treatment effectiveness.
DOI 10.1371/journal.pcbi.1009370