{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,8]],"date-time":"2025-10-08T16:25:55Z","timestamp":1759940755634},"reference-count":32,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2005,12,1]],"date-time":"2005-12-01T00:00:00Z","timestamp":1133395200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2005,12]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Mucopolysaccharidosis I (MPS I) is a lysosomal disorder characterized by a deficiency of the enzyme \u03b1\u2010<jats:sc>L<\/jats:sc>\u2010iduronidase (IDUA), which is responsible for the degradation of glycosaminoglycans (GAGs). This deficiency leads to the accumulation of dermatan and heparan sulphate in lysosomes. Presently available treatments include bone marrow transplantation and enzyme replacement therapies, both of which are limited in their effects. In this work, knockout (KO) MPS I mice were treated with a nonviral vector containing the human <jats:italic>IDUA<\/jats:italic> cDNA. KO mice were transfected by hydrodynamic injection of pRIDUA in the caudal vein (i.v., <jats:italic>n<\/jats:italic> = 3) or by intraperitoneal injection of pRIDUA\/Superfect complexes (i.p., <jats:italic>n<\/jats:italic> = 3). GAG concentration and IDUA activity were analysed in the kidneys, spleen, lungs, brain and liver. The expression of IDUA in the organs of i.v.\u2010 and i.p.\u2010treated mice was also analysed by real\u2010time reverse\u2010transcription (RT) PCR and compared by relative quantification. The concentration of GAGs in the organs differed between KO and wild\u2010type mice. In the spleen and liver, GAG levels were lower in i.v.\u2010 and i.p.\u2010treated KO mice than in control nontreated animals. Real\u2010time RT\u2010PCR showed that the transgene is expressed in all the analysed organs of i.p.\u2010 and i.v.\u2010treated KO mice. Enzyme activity was similarly observed in all the organs analysed. Our data suggest that this kind of transfection may be a useful tool for studies of nonviral protocols for gene therapy of MPS.<\/jats:p>","DOI":"10.1007\/s10545-005-0070-5","type":"journal-article","created":{"date-parts":[[2006,1,25]],"date-time":"2006-01-25T09:35:29Z","timestamp":1138181729000},"page":"1035-1043","source":"Crossref","is-referenced-by-count":19,"title":["Nonviral <i>in vivo<\/i> gene transfer in the mucopolysaccharidosis I murine model"],"prefix":"10.1002","volume":"28","author":[{"given":"M.","family":"Camassola","sequence":"first","affiliation":[{"name":"Genetics Department Universidade Federal do Rio Grande do Sul Caixa Postal 15053 Porto Alegre RS 91501\u2010900 Brasil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"L. 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