{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,29]],"date-time":"2026-05-29T20:51:35Z","timestamp":1780087895139,"version":"3.54.0"},"reference-count":24,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2011,9,26]],"date-time":"2011-09-26T00:00:00Z","timestamp":1316995200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2011,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction:<\/jats:title><jats:p>Enzyme replacement therapy (ERT) in ultra\u2010orphan Pompe disease generates anti\u2010rhGAA antibodies, which may interfere with efficacy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods:<\/jats:title><jats:p>rhGAA\u2010specific T\u2010cell responses were examined at different time\u2010points in 6 Hungarian patients treated with rhGAA and compared with 1 untreated patient and 5 healthy controls.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results:<\/jats:title><jats:p>The <jats:italic>ex vivo<\/jats:italic> percentage of activated T cells was increased in treated patients. rhGAA stimulation in vitro generated a dose\u2010dependent increase in intracellular interferon\u2010gamma (IFN\u2010\u03b3) expression in CD4<jats:sup>+<\/jats:sup> and CD8<jats:sup>+<\/jats:sup> T cells. Isolated CD4<jats:sup>+<\/jats:sup> and CD8<jats:sup>+<\/jats:sup> T cells produced increased amounts of IFN\u2010\u03b3 and tumor necrosis factor\u2010alpha (TNF\u2010\u03b1) in half of the patients after <jats:italic>in vitro<\/jats:italic> stimulation with rhGAA, whereas interleukin (IL)\u20104, IL\u20106, and IL\u201017 levels were not elevated. Expression of cytotoxic FasL and perforin molecules by natural killer (NK), NKT\u2010like, and CD8<jats:sup>+<\/jats:sup> T cells were not increased <jats:italic>ex vivo<\/jats:italic>.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions:<\/jats:title><jats:p>We found that enzyme replacement therapy (ERT) induces pro\u2010inflammatory T\u2010cell responses in addition to the antibody response in Pompe disease. Muscle Nerve, 2011<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.22136","type":"journal-article","created":{"date-parts":[[2011,4,18]],"date-time":"2011-04-18T14:12:59Z","timestamp":1303135979000},"page":"720-726","source":"Crossref","is-referenced-by-count":11,"title":["Enzyme replacement therapy induces T\u2010cell responses in late\u2010onset Pompe disease"],"prefix":"10.1002","volume":"44","author":[{"given":"Miklos","family":"Banati","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zsolt","family":"Hosszu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anita","family":"Trauninger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laszlo","family":"Szereday","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zsolt","family":"Illes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2011,9,26]]},"reference":[{"key":"e_1_2_6_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)61555-X"},{"key":"e_1_2_6_3_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.gt.3302314"},{"key":"e_1_2_6_4_2","doi-asserted-by":"publisher","DOI":"10.1086\/522236"},{"key":"e_1_2_6_5_2","doi-asserted-by":"publisher","DOI":"10.1542\/peds.100.6.e11"},{"key":"e_1_2_6_6_2","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1182\/blood.V90.1.43.43_43_48","article-title":"Enzyme therapy in Gaucher disease type I: effect of neutralizing antibodies to acid \u03b2\u2010glucosidase","volume":"90","author":"Ponce E","year":"1997","journal-title":"Blood"},{"key":"e_1_2_6_7_2","doi-asserted-by":"publisher","DOI":"10.1097\/00125817-200103000-00008"},{"key":"e_1_2_6_8_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2006.02.035"},{"key":"e_1_2_6_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2004.10.009"},{"key":"e_1_2_6_10_2","doi-asserted-by":"publisher","DOI":"10.1542\/peds.113.5.e448"},{"key":"e_1_2_6_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(03)13311-9"},{"key":"e_1_2_6_12_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-3083.2009.02308.x"},{"key":"e_1_2_6_13_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-05-078154"},{"key":"e_1_2_6_14_2","doi-asserted-by":"publisher","DOI":"10.4049\/jimmunol.167.3.1245"},{"key":"e_1_2_6_15_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.7518614"},{"key":"e_1_2_6_16_2","doi-asserted-by":"publisher","DOI":"10.1038\/370650a0"},{"key":"e_1_2_6_17_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0007980"},{"key":"e_1_2_6_18_2","doi-asserted-by":"publisher","DOI":"10.1034\/j.1600-065X.2003.00051.x"},{"key":"e_1_2_6_19_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2009.08.003"},{"key":"e_1_2_6_20_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2008.03.008"},{"key":"e_1_2_6_21_2","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2006-2156"},{"key":"e_1_2_6_22_2","doi-asserted-by":"publisher","DOI":"10.1001\/archneurol.2010.21"},{"key":"e_1_2_6_23_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa044397"},{"key":"e_1_2_6_24_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2249.2010.04258.x"},{"key":"e_1_2_6_25_2","doi-asserted-by":"publisher","DOI":"10.1160\/TH06-09-0519"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fmus.22136","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fmus.22136","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.22136","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,10,9]],"date-time":"2023-10-09T16:39:37Z","timestamp":1696869577000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.22136"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2011,9,26]]},"references-count":24,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2011,11]]}},"alternative-id":["10.1002\/mus.22136"],"URL":"https:\/\/doi.org\/10.1002\/mus.22136","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2011,9,26]]}}}