Concepedia

Carnitine is a pharmacological allosteric chaperone of the human lysosomal <i>α</i>-glucosidase

Roberta Iacono, Nadia Minopoli, Maria Carmina Ferrara, Antonietta Tarallo, Carla Damiano, Caterina Porto, Sandra Strollo, V. Roig-Zamboni, Gianfranco Peluso, Gerlind Sulzenbacher,

Journal of Enzyme Inhibition and Medicinal Chemistry · 2021 · 10 citations · 47 references

DOIFull text

Open access

Concepts

Abstract

-glucosidase activity in fibroblasts from three Pompe patients with added rhGAA. This synergistic effect of L-carnitine and rhGAA has the potential to be translated into improved therapeutic efficacy of ERT in Pompe disease.

References

47

The carbohydrate-active enzymes database (CAZy) in 2013

Vincent Lombard, Hemalatha Golaconda Ramulu, Élodie Drula et al. · Nucleic Acids Research · 2013 · 6.5K citations · Full text

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The use of differential scanning fluorimetry to detect ligand interactions that promote protein stability

F. Niesen, H. Berglund, Masoud Vedadi · Nature Protocols · 2007 · 2.4K citations

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Recombinant human acid α-glucosidase

Priya S. Kishnani, Deya Corzo, Marc Nicolino et al. · Neurology · 2006 · 772 citations · Full text

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A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

Ans T. van der Ploeg, Paula R. Clemens, Deyanira Corzo et al. · New England Journal of Medicine · 2010 · 747 citations · Full text

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Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

Priya S. Kishnani, Paula Goldenberg, Stephanie DeArmey et al. · Molecular Genetics and Metabolism · 2009 · 379 citations

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