{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T12:15:20Z","timestamp":1774786520692,"version":"3.50.1"},"reference-count":36,"publisher":"Portland Press Ltd.","issue":"2","content-domain":{"domain":["portlandpress.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2005,6,1]]},"abstract":"<jats:p>The Sanfilippo syndrome type B (mucopolysaccharidosis IIIB) is an autosomal recessive disorder due to mutations in the gene encoding NAGLU (\u03b1-N-acetylglucosaminidase), one of the enzymes required for the degradation of the GAG (glycosaminoglycan) heparan sulphate. No therapy exists for affected patients. We have shown previously the efficacy of lentiviral-NAGLU-mediated gene transfer in correcting in vitro the defect on fibroblasts of patients. In the present study, we tested the therapy in vivo on a knockout mouse model using intravenous injections. Mice (8\u201310\u00a0weeks old) were injected with one of the lentiviral doses through the tail vein and analysed 1\u00a0month after treatment. A single injection of lentiviral-NAGLU vector resulted in transgene expression in liver, spleen, lung and heart of treated mice, with the highest level reached in liver and spleen. Expression of 1% normal NAGLU activity in liver resulted in a 77% decrease in the GAG content; more remarkably, an expression of 0.16% normal activity in lung was capable of decreasing the GAG level by 29%. Long-term (6\u00a0months) follow up of the gene therapy revealed that the viral genome integration persisted in the target tissues, although the real-time PCR analysis showed a decrease in the vector DNA content with time. Interestingly, the decrease in GAG levels was maintained in liver, spleen, lung and heart of treated mice. These results show the promising potential and the limitations of lentiviral-NAGLU vector to deliver the human NAGLU gene in vivo.<\/jats:p>","DOI":"10.1042\/bj20041702","type":"journal-article","created":{"date-parts":[[2005,6,7]],"date-time":"2005-06-07T09:42:14Z","timestamp":1118137334000},"page":"639-646","update-policy":"https:\/\/doi.org\/10.1042\/crossmark_policy","source":"Crossref","is-referenced-by-count":51,"title":["Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector"],"prefix":"10.1042","volume":"388","author":[{"given":"Paola","family":"Di\u00a0NATALE","sequence":"first","affiliation":[{"name":"Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Via S. Pansini n. 5, 80131 Naples, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Carmela","family":"Di\u00a0DOMENICO","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Via S. Pansini n. 5, 80131 Naples, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nadia","family":"GARGIULO","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Via S. Pansini n. 5, 80131 Naples, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sigismondo","family":"CASTALDO","sequence":"additional","affiliation":[{"name":"Center for Animal Experimentation, Cardarelli Hospital, Naples, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Enrico\u00a0GONZALEZ\u00a0Y","family":"REYERO","sequence":"additional","affiliation":[{"name":"Center for Animal Experimentation, Cardarelli Hospital, Naples, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pratibha","family":"MITHBAOKAR","sequence":"additional","affiliation":[{"name":"Stazione Zoologica Anton Dohrn, Naples, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mario","family":"De\u00a0FELICE","sequence":"additional","affiliation":[{"name":"Stazione Zoologica Anton Dohrn, Naples, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Antonia","family":"FOLLENZI","sequence":"additional","affiliation":[{"name":"HSR-Telethon Institute for Gene Therapy, H. San Raffaele Scientific Institute, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Luigi","family":"NALDINI","sequence":"additional","affiliation":[{"name":"HSR-Telethon Institute for Gene Therapy, H. San Raffaele Scientific Institute, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Guglielmo\u00a0R.\u00a0D.","family":"VILLANI","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Via S. 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