Pharmacological chaperone therapy for lysosomal storage disorders - leveraging aspects of the folding pathway to maximize activity of misfolded mutant proteins.
Fan
Jian-Qiang
JQ
Pfantastic Medical Research Institute, Cresskill, NJ, USA.
D006801:Humans; D016464:Lysosomal Storage Diseases / Q000188:drug therapy* / Q000378:metabolism; D009154:Mutation; D017510:Protein Folding