A Chaperone Enhances Blood α-Glucosidase Activity in Pompe Disease Patients Treated With Enzyme Replacement Therapy

Enzyme replacement therapy is currently the only approved treatment for Pompe disease, due to acid α-glucosidase deficiency. Clinical efficacy of this approach is variable, and more effective therapies are needed. We showed in preclinical studies that chaperones stabilize the recombinant enzyme used...

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Opis bibliograficzny
Wydane w:Mol Ther
Główni autorzy: Parenti, Giancarlo, Fecarotta, Simona, la Marca, Giancarlo, Rossi, Barbara, Ascione, Serena, Donati, Maria Alice, Morandi, Lucia Ovidia, Ravaglia, Sabrina, Pichiecchio, Anna, Ombrone, Daniela, Sacchini, Michele, Pasanisi, Maria Barbara, De Filippi, Paola, Danesino, Cesare, Della Casa, Roberto, Romano, Alfonso, Mollica, Carmine, Rosa, Margherita, Agovino, Teresa, Nusco, Edoardo, Porto, Caterina, Andria, Generoso
Format: Artigo
Język:Inglês
Wydane: Nature Publishing Group 2014
Hasła przedmiotowe:
Original Article
Dostęp online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4429731/
https://ncbi.nlm.nih.gov/pubmed/25052852
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.138
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