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In addition to glycogen storage, secondary dysregulation of cellular functions, such as autophagy and oxidative stress, contributes to the disease pathophysiology. We have tested whether oxidative stress impacts on enzyme replacement therapy with recombinant human alpha\u2010glucosidase (rhGAA), currently the standard of care for Pompe disease patients, and whether correction of oxidative stress may be beneficial for rhGAA therapy. We found elevated oxidative stress levels in tissues from the Pompe disease murine model and in patients\u2019 cells. In cells, stress levels inversely correlated with the ability of rhGAA to correct the enzymatic deficiency. Antioxidants (N\u2010acetylcysteine, idebenone, resveratrol, edaravone) improved alpha\u2010glucosidase activity in rhGAA\u2010treated cells, enhanced enzyme processing, and improved mannose\u20106\u2010phosphate receptor localization. When co\u2010administered with rhGAA, antioxidants improved alpha\u2010glucosidase activity in tissues from the Pompe disease mouse model. These results indicate that oxidative stress impacts on the efficacy of enzyme replacement therapy in Pompe disease and that manipulation of secondary abnormalities may represent a strategy to improve the efficacy of therapies for this disorder.<\/jats:p>","DOI":"10.15252\/emmm.202114434","type":"journal-article","created":{"date-parts":[[2021,10,7]],"date-time":"2021-10-07T16:31:04Z","timestamp":1633624264000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":26,"title":["Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease"],"prefix":"10.1038","volume":"13","author":[{"given":"Antonietta","family":"Tarallo","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla","family":"Damiano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5397-2928","authenticated-orcid":false,"given":"Sandra","family":"Strollo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1675-0538","authenticated-orcid":false,"given":"Nadia","family":"Minopoli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2325-0913","authenticated-orcid":false,"given":"Alessia","family":"Indrieri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Polishchuk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Zappa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edoardo","family":"Nusco","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simona","family":"Fecarotta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caterina","family":"Porto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcella","family":"Coletta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3586-4322","authenticated-orcid":false,"given":"Roberta","family":"Iacono","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marco","family":"Moracci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7698-1955","authenticated-orcid":false,"given":"Roman","family":"Polishchuk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7347-2645","authenticated-orcid":false,"given":"Diego Luis","family":"Medina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paola","family":"Imbimbo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daria Maria","family":"Monti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0053-3061","authenticated-orcid":false,"given":"Maria Antonietta","family":"De Matteis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6287-5748","authenticated-orcid":false,"given":"Giancarlo","family":"Parenti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,10,4]]},"reference":[{"key":"BFEMMM202114434_CR1","doi-asserted-by":"crossref","first-page":"2652","DOI":"10.1111\/j.1582-4934.2011.01271.x","volume":"15","author":"A Arciello","year":"2011","unstructured":"Arciello A, De Marco N, Del Giudice R, Guglielmi F, Pucci P, Relini A, Monti DM, Piccoli R (2011) Insights into the fate of the N\u2010terminal amyloidogenic polypeptide of ApoA\u2010I in cultured target cells. 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