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1,6--Cyclophellitol Cyclosulfamidate Is a Bona Fide Lysosomal α-Glucosidase Stabilizer for the Treatment of Pompe Disease.
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Pharmacological Chaperone Therapy for Pompe Disease.
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Carnitine is a pharmacological allosteric chaperone of the human lysosomal -glucosidase.
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Second-Generation Pharmacological Chaperones: Beyond Inhibitors.
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Non-iminosugar glucocerebrosidase small molecule chaperones.
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A high throughput glucocerebrosidase assay using the natural substrate glucosylceramide.
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本文引用的文献

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Pharmacological Enhancement of Mutated α-Glucosidase Activity in Fibroblasts from Patients with Pompe Disease.
Mol Ther. 2007 Mar;15(3):508-514. doi: 10.1038/sj.mt.6300074. Epub 2016 Dec 8.
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A new resorufin-based alpha-glucosidase assay for high-throughput screening.
Anal Biochem. 2009 Jul 1;390(1):79-84. doi: 10.1016/j.ab.2009.04.010. Epub 2009 Apr 14.
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Active-site-specific chaperone therapy for Fabry disease. Yin and Yang of enzyme inhibitors.
FEBS J. 2007 Oct;274(19):4962-71. doi: 10.1111/j.1742-4658.2007.06041.x.
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Chemical chaperones improve transport and enhance stability of mutant alpha-glucosidases in glycogen storage disease type II.
Mol Genet Metab. 2007 Jan;90(1):49-57. doi: 10.1016/j.ymgme.2006.09.010. Epub 2006 Nov 13.
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New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy.
Hum Genet. 2007 Mar;121(1):1-22. doi: 10.1007/s00439-006-0280-4. Epub 2006 Nov 7.
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Characterization of human saposins by NMR spectroscopy.
Biochemistry. 2006 Apr 25;45(16):5206-16. doi: 10.1021/bi051944+.
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Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease).
Curr Mol Med. 2002 Mar;2(2):145-66. doi: 10.2174/1566524024605789.

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