N-Butyl-l-deoxynojirimycin (l-NBDNJ): Synthesis of an Allosteric Enhancer of &#945;-Glucosidase Activity for the Treatment of Pompe Disease.

D'Alonzo, Daniele; De Fenza, Maria; Porto, Caterina; Iacono, Roberta; Huebecker, Mylene; Cobucci-Ponzano, Beatrice; Priestman, David A; Platt, Frances; Parenti, Giancarlo; Moracci, Marco; Palumbo, Giovanni; Guaragna, Annalisa

N-Butyl-l-deoxynojirimycin (l-NBDNJ): Synthesis of an Allosteric Enhancer of α-Glucosidase Activity for the Treatment of Pompe Disease.

D'Alonzo, Daniele; De Fenza, Maria; Porto, Caterina; Iacono, Roberta; Huebecker, Mylene; Cobucci-Ponzano, Beatrice; Priestman, David A; Platt, Frances; Parenti, Giancarlo; Moracci, Marco; Palumbo, Giovanni; Guaragna, Annalisa.

Affiliation

D'Alonzo D; Department of Chemical Sciences, Università degli Studi di Napoli Federico II , via Cintia, 80126 Napoli, Italy.
De Fenza M; Department of Chemical Sciences, Università degli Studi di Napoli Federico II , via Cintia, 80126 Napoli, Italy.
Porto C; Department of Translational Medical Sciences, Section of Pediatrics, Università degli Studi di Napoli Federico II , Via S. Pansini 5, 80131 Napoli, Italy.
Iacono R; Institute of Biosciences and Bioresources, Consiglio Nazionale delle Ricerche , Via P. Castellino 111, 80131 Napoli, Italy.
Huebecker M; Department of Pharmacology, University of Oxford , Mansfield Road, Oxford OX1 3QT, U.K.
Cobucci-Ponzano B; Institute of Biosciences and Bioresources, Consiglio Nazionale delle Ricerche , Via P. Castellino 111, 80131 Napoli, Italy.
Priestman DA; Department of Pharmacology, University of Oxford , Mansfield Road, Oxford OX1 3QT, U.K.
Platt F; Department of Pharmacology, University of Oxford , Mansfield Road, Oxford OX1 3QT, U.K.
Parenti G; Department of Translational Medical Sciences, Section of Pediatrics, Università degli Studi di Napoli Federico II , Via S. Pansini 5, 80131 Napoli, Italy.
Moracci M; Telethon Institute of Genetics and Medicine , Via Campi Flegrei 34, 80078 Pozzuoli, Italy.
Palumbo G; Institute of Biosciences and Bioresources, Consiglio Nazionale delle Ricerche , Via P. Castellino 111, 80131 Napoli, Italy.
Guaragna A; Department of Biology, Università degli Studi di Napoli Federico II , via Cintia, 80126 Napoli, Italy.

J Med Chem ; 60(23): 9462-9469, 2017 12 14.

Article in En | MEDLINE | ID: mdl-29112434

Subject(s)

1-Deoxynojirimycin/analogs & derivatives; Enzyme Activation/drug effects; Fibroblasts/drug effects; Glycogen Storage Disease Type II/drug therapy; alpha-Glucosidases/metabolism; 1-Deoxynojirimycin/chemical synthesis; 1-Deoxynojirimycin/chemistry; 1-Deoxynojirimycin/pharmacology; Allosteric Regulation/drug effects; Cell Line; Enzyme Inhibitors/chemical synthesis; Enzyme Inhibitors/chemistry; Enzyme Inhibitors/pharmacology; Fibroblasts/enzymology; Fibroblasts/metabolism; Glycogen Storage Disease Type II/enzymology; Glycogen Storage Disease Type II/metabolism; Humans; Lysosomes/drug effects; Lysosomes/enzymology; Lysosomes/metabolism; Models, Molecular; Stereoisomerism

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II / 1-Deoxynojirimycin / Enzyme Activation / Alpha-Glucosidases / Fibroblasts Limits: Humans Language: En Journal: J Med Chem Journal subject: QUIMICA Year: 2017 Document type: Article Affiliation country: Italy Country of publication: United States

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