Targeted delivery of pharmacological chaperones for Gaucher disease to macrophages by a mannosylated cyclodextrin carrier

Gaucher disease (GD) is a rare monogenetic disorder leading to dysfunction of acid β-glucosidase (β-glucocerebrosidase; GCase) and accumulation of glucosylceramide in lysosomes, especially in macrophages (Gaucher cells). Many of the mutations at the origin of GD do not impair the catalytic activity...

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Veröffentlicht in:	Organic & biomolecular chemistry 2014-04, Vol.12 (14), p.2289-2301
Hauptverfasser:	Rodríguez-Lavado, Julio, de la Mata, Mario, Jiménez-Blanco, José L, García-Moreno, M Isabel, Benito, Juan M, Díaz-Quintana, Antonio, Sánchez-Alcázar, José A, Higaki, Katsumi, Nanba, Eiji, Ohno, Kousaku, Suzuki, Yoshiyuki, Ortiz Mellet, Carmen, García Fernández, José M
Format:	Artikel
Sprache:	eng
Schlagworte:	beta-Cyclodextrins - administration & dosage beta-Cyclodextrins - chemistry Carbohydrate Conformation Drug Carriers - administration & dosage Drug Carriers - chemistry Drug Delivery Systems Gaucher Disease - drug therapy Gaucher Disease - pathology Humans Macrophages - drug effects Macrophages - metabolism Microscopy, Fluorescence Molecular Chaperones - administration & dosage Molecular Chaperones - pharmacology Molecular Chaperones - therapeutic use
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