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, Beatrice Cobucci‐Ponzano, Giancarlo Parenti, Marco Moracci
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.
Topics & Concepts
Allosteric regulationEnzymeEnzyme replacement therapyChaperone (clinical)Chemical chaperoneChemistryBiochemistryCarnitinePharmacologySmall moleculeBiologyDiseaseMedicineInternal medicineUnfolded protein responseGenePathologyLysosomal Storage Disorders ResearchCarbohydrate Chemistry and SynthesisBiochemical and Molecular Research