The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts.

Porto, Caterina; Cardone, Monica; Fontana, Federica; Rossi, Barbara; Tuzzi, Maria Rosaria; Tarallo, Antonietta; Barone, Maria Vittoria; Andria, Generoso; Parenti, Giancarlo

Porto, Caterina; Cardone, Monica; Fontana, Federica; Rossi, Barbara; Tuzzi, Maria Rosaria; Tarallo, Antonietta; Barone, Maria Vittoria; Andria, Generoso; Parenti, Giancarlo.

Affiliation

Porto C; Department of Pediatrics, Federico II University, Naples, Italy.

Mol Ther ; 17(6): 964-71, 2009 Jun.

Article in En | MEDLINE | ID: mdl-19293774

Subject(s)

1-Deoxynojirimycin/analogs & derivatives; Enzyme Inhibitors/therapeutic use; Fibroblasts/drug effects; Glycogen Storage Disease Type II/drug therapy; 1-Deoxynojirimycin/pharmacology; 1-Deoxynojirimycin/therapeutic use; Animals; Biological Transport/drug effects; Blotting, Western; Cell Line; Drug Stability; Enzyme Inhibitors/pharmacology; Fibroblasts/pathology; Humans; Lysosomes/metabolism; Mice; Microscopy, Confocal; alpha-Galactosidase/administration & dosage; alpha-Galactosidase/metabolism; alpha-Galactosidase/pharmacology; alpha-Galactosidase/therapeutic use

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II / 1-Deoxynojirimycin / Enzyme Inhibitors / Fibroblasts Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2009 Document type: Article Affiliation country: Italy Country of publication: United States

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