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Here we tested the potential of a helper-dependent CAV-2 vector expressing \u03b2-glucuronidase (HD-RIGIE) in a mouse model of mucopolysaccharidosis type VII (MPS VII), a lysosomal storage disease caused by deficiency in \u03b2-glucuronidase activity. MPS VII leads to glycosaminoglycan accumulation into enlarged vesicles in peripheral tissues and the CNS, resulting in peripheral and neuronal dysfunction. After intracranial administration of HD-RIGIE, we show long-term expression of \u03b2-glucuronidase that led to correction of neuropathology around the injection site and in distal areas. This phenotypic correction correlated with a decrease in secondary-elevated lysosomal enzyme activity and glycosaminoglycan levels, consistent with global biochemical correction. Moreover, HD-RIGIE-treated mice show significant cognitive improvement. Thus, injections of HD-CAV-2 vectors in the brain allow a global and sustained expression and may have implications for brain therapy in patients with lysosomal storage disease.<\/jats:p>","DOI":"10.1089\/hum.2013.152","type":"journal-article","created":{"date-parts":[[2013,12,3]],"date-time":"2013-12-03T17:47:50Z","timestamp":1386092870000},"page":"199-211","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":29,"title":["Central Nervous System Delivery of Helper-Dependent Canine Adenovirus Corrects Neuropathology and Behavior in Mucopolysaccharidosis Type VII Mice"],"prefix":"10.1177","volume":"25","author":[{"given":"Lorena","family":"Ariza","sequence":"first","affiliation":[{"name":"Universitat Aut\u00f2noma de Barcelona","place":["Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lydia","family":"Gim\u00e9nez-Llort","sequence":"additional","affiliation":[{"name":"Universitat Aut\u00f2noma de 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