Pharmacologic chaperoning as a strategy to treat Gaucher disease

We briefly introduce the most common lysosomal storage disorder, Gaucher disease, concisely describe the Food and Drug Administration approved strategies to ameliorate Gaucher disease, and then outline the emerging pharmacologic chaperone strategy that offers the promise to remedy this malady.

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Bibliographische Detailangaben
Veröffentlicht in:	The FEBS journal 2007-10, Vol.274 (19), p.4944-4950
Hauptverfasser:	Yu, Zhanqian, Sawkar, Anu R, Kelly, Jeffery W
Format:	Artikel
Sprache:	eng
Schlagworte:	endoplasmic reticulum‐associated degradation folding Gaucher disease Gaucher Disease - drug therapy Gaucher Disease - genetics Glucosylceramidase - chemistry Glucosylceramidase - genetics Glucosylceramidase - therapeutic use Humans Models, Molecular neuropathic Gaucher disease pharmacologic chaperone Protein Conformation traficking type 2 Gaucher disease type 3 Gaucher disease
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Beschreibung
Zusammenfassung:	We briefly introduce the most common lysosomal storage disorder, Gaucher disease, concisely describe the Food and Drug Administration approved strategies to ameliorate Gaucher disease, and then outline the emerging pharmacologic chaperone strategy that offers the promise to remedy this malady.
ISSN:	1742-464X 1742-4658
DOI:	10.1111/j.1742-4658.2007.06042.x