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Although several therapeutic approaches have been applied to the murine model of the disease, no effective therapy is available for patients. In this study, we used the lentiviral\u2010NAGLU vector to deliver the functional human NAGLU gene into the brain of young adult MPS IIIB mice. We report the restoration of active enzyme with a sustained expression throughout a large portion of the brain, and a significantly improved behavioral performance of treated animals. Moreover, we analyzed the effect of therapy on the expression profile of some genes related to neurotrophic signaling molecules and inflammatory cytokines previously found altered in MPS IIIB mice. At 1 month from treatment, the level of cerebellin 1 (Cbln1) was decreased while the brain\u2010derived neurotrophic factor (Bdnf) expression was increased, both reaching normal values. At 6 months from treatment a significant reduction in the expression of all the inflammation\u2010 and oxidative stress\u2010related genes was observed, as well as the maintenance of the correction of the Bdnf gene expression. These results indicate that NAGLU delivery from intracerebral sources has the capacity to alleviate most disease manifestations in MPS IIIB mice; furthermore, Bdnf might be a response\u2010to\u2010therapy biomarker for MPS IIIB. \u00a9 2009 Wiley\u2010Liss, Inc.<\/jats:p>","DOI":"10.1002\/ajmg.a.32861","type":"journal-article","created":{"date-parts":[[2009,5,16]],"date-time":"2009-05-16T00:14:19Z","timestamp":1242432859000},"page":"1209-1218","source":"Crossref","is-referenced-by-count":26,"title":["Intracranial gene delivery of LV\u2010NAGLU vector corrects neuropathology in murine MPS IIIB"],"prefix":"10.1002","volume":"149A","author":[{"given":"Carmela","family":"Di Domenico","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Guglielmo R.D.","family":"Villani","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Daniele","family":"Di Napoli","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Edoardo","family":"Nusco","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gaetano","family":"Cal\u00ec","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lucio","family":"Nitsch","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paola","family":"Di Natale","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2009,5,15]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1089\/10430340252899019"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0306-4522(03)00564-5"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-006-0280-4"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2005.16.1133"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1089\/10430340050015194"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1012283426530"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0603765103"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.expneurol.2007.10.009"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1038\/85454"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0404180101"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2006.05.005"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.3558-04.2004"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.20150"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2005.08.020"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1042\/BJ20041702"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.1042\/BA20070093"},{"key":"e_1_2_6_18_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0409072102"},{"key":"e_1_2_6_19_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1025177411938"},{"key":"e_1_2_6_20_1","first-page":"259","article-title":"HIV\u2010based vectors. 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