Pharmacologic chaperoning as a strategy to treat Gaucher disease.
We briefly introduce the most common lysosomal storage disorder, Gaucher disease, concisely describe the Food and Drug Administration approved strategies to ameliorate Gaucher disease, and then outline the emerging pharmacologic chaperone strategy that offers the promise to remedy this malady.
Yu
Zhanqian
Z
Department of Chemistry and The Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA 92037, USA.
Sawkar
Anu R
AR
Kelly
Jeffery W
JW
D005776:Gaucher Disease / Q000188:drug therapy* / Q000235:genetics; D005962:Glucosylceramidase / Q000737:chemistry / Q000235:genetics / Q000627:therapeutic use; D006801:Humans; D008958:Models, Molecular; D011487:Protein Conformation
D005962:Glucosylceramidase