Lysosomal delivery of therapeutic enzymes in cell models of Fabry disease

The success of enzymatic replacement in Gaucher disease has stimulated development of targeted protein replacement for other lysosomal disorders, including Anderson-Fabry disease, which causes fatal cardiac, cerebrovascular and renal injury: deficiency of lysosomal α-Galactosidase A induces accumula...

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Veröffentlicht in:	Journal of inherited metabolic disease 2012-11, Vol.35 (6), p.1107-1117
Hauptverfasser:	Marchesan, D., Cox, T. M., Deegan, P. B.
Format:	Artikel
Sprache:	eng
Schlagworte:	alpha-Galactosidase - administration & dosage alpha-Galactosidase - pharmacokinetics Biochemistry Biological and medical sciences Cell Line Cells, Cultured Drug Delivery Systems - methods Endothelial Cells - enzymology Enzyme Replacement Therapy - methods Errors of metabolism Fabry Disease - drug therapy Fabry Disease - enzymology Fibroblasts - enzymology Human Genetics Humans Internal Medicine Isoenzymes - administration & dosage Isoenzymes - pharmacokinetics Lipids (lysosomal enzyme disorders, storage diseases) Lysosomes - enzymology Medical genetics Medical sciences Medicine Medicine & Public Health Metabolic Diseases Microscopy, Confocal Models, Biological Original Article Pediatrics Receptor, IGF Type 2 - metabolism Recombinant Proteins - administration & dosage Recombinant Proteins - pharmacokinetics
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