Cell-based screening of active-site specific chaperone for the treatment of Fabry disease.
Department of Human Genetics, Mount Sinai School of Medicine, Fifth Avenue at 100 Street, New York, New York 10029, USA.
Jian-Qiang
JQ
Fan
Satoshi
S
Ishii
D000818:Animals; D001483:Base Sequence; D001693:Biological Transport, Active; D019556:COS Cells; D020134:Catalytic Domain; D018076:DNA, Complementary / Q000235:genetics; D004353:Drug Evaluation, Preclinical / Q000379:methods*; D004721:Endoplasmic Reticulum / Q000201:enzymology; D000795:Fabry Disease / Q000188:drug therapy* / Q000201:enzymology / Q000235:genetics; D006801:Humans; D007150:Immunohistochemistry; D066298:In Vitro Techniques; D018832:Molecular Chaperones / Q000627:therapeutic use*; D009154:Mutation; D017510:Protein Folding; D011994:Recombinant Proteins / Q000737:chemistry / Q000235:genetics / Q000378:metabolism; D014162:Transfection; D000519:alpha-Galactosidase / Q000737:chemistry / Q000235:genetics / Q000378:metabolism
D018076:DNA, Complementary; D018832:Molecular Chaperones; D011994:Recombinant Proteins; D000519:alpha-Galactosidase