Enzyme Enhancers for the Treatment of Fabry and Pompe Disease

Lysosomal storage disorders (LSD) are a group of heterogeneous diseases caused by compromised enzyme function leading to multiple organ failure. Therapeutic approaches involve enzyme replacement (ERT), which is effective for a substantial fraction of patients. However, there are still concerns about...

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Veröffentlicht in:	Molecular therapy 2015-03, Vol.23 (3), p.456-464
Hauptverfasser:	Lukas, Jan, Pockrandt, Anne-Marie, Seemann, Susanne, Sharif, Muhammad, Runge, Franziska, Pohlers, Susann, Zheng, Chaonan, Gläser, Anne, Beller, Matthias, Rolfs, Arndt, Giese, Anne-Katrin
Format:	Artikel
Sprache:	eng
Schlagworte:	1-Deoxynojirimycin - analogs & derivatives 1-Deoxynojirimycin - pharmacology Acetylcysteine - analogs & derivatives Acetylcysteine - pharmacology alpha-Galactosidase - genetics alpha-Galactosidase - metabolism alpha-Glucosidases - genetics alpha-Glucosidases - metabolism Ambroxol - pharmacology Antibodies Bezafibrate - pharmacology Disease Endoplasmic reticulum Enzyme Activators - pharmacology Enzymes Fabry Disease - drug therapy Fabry Disease - enzymology Gene Expression Glycogen Storage Disease Type II - drug therapy Glycogen Storage Disease Type II - enzymology HEK293 Cells Humans Leupeptins - pharmacology LSD Lysergic acid diethylamide Lysosomes - drug effects Lysosomes - metabolism Mutation Original Pioglitazone Plasmids - chemistry Plasmids - metabolism Proteasome Endopeptidase Complex - drug effects Proteasome Endopeptidase Complex - metabolism Proteasome Inhibitors - pharmacology Protein Stability Recombinant Proteins - genetics Recombinant Proteins - metabolism Thiazolidinediones - pharmacology Transfection
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