Please use this identifier to cite or link to this item: http://hdl.handle.net/10553/46097
DC FieldValueLanguage
dc.contributor.authorSalido, Eduardoen_US
dc.contributor.authorRodriguez-Pena, Marisolen_US
dc.contributor.authorSantana, Alfredoen_US
dc.contributor.authorBeattie, Stuart G.en_US
dc.contributor.authorPetry, Haralden_US
dc.contributor.authorTorres, Armandoen_US
dc.date.accessioned2018-11-23T01:21:09Z-
dc.date.available2018-11-23T01:21:09Z-
dc.date.issued2011en_US
dc.identifier.issn1525-0016en_US
dc.identifier.urihttp://hdl.handle.net/10553/46097-
dc.description.abstractPrimary hyperoxaluria type I (PH1) is an inborn error of metabolism caused by deficiency of the hepatic enzyme alanine-glyoxylate aminotransferase (AGXT or AGT) which leads to overproduction of oxalate by the liver and subsequent urolithiasis and renal failure. The current therapy largely depends on liver transplantation, which is associated with significant morbidity and mortality. To explore an alternative treatment, we used somatic gene transfer in a mouse genetic model for PH1 (Agxt1KO). Recombinant adeno-associated virus (AAV) vectors containing the human AGXT complementary DNA (cDNA) were pseudotyped with capsids from either serotype 8 or 5, and delivered to the livers of Agxt1KO mice via the tail vein. Both AAV8-AGXT and AAV5-AGXT vectors were able to reduce oxaluria to normal levels. In addition, treated mice showed blunted increase of oxaluria after challenge with ethylene glycol (EG), a glyoxylate precursor. In mice, AGT enzyme activity in whole liver extracts were restored to normal without hepatic toxicity nor immunogenicity for the 50 day follow-up. In summary, this study demonstrates the correction of primary hyperoxaluria in mice treated with either AAV5 or AAV8 vectors.en_US
dc.languageengen_US
dc.relation.ispartofMolecular Therapyen_US
dc.sourceMolecular Therapy [ISSN 1525-0016],v. 19, p. 870-875en_US
dc.subject32 Ciencias médicasen_US
dc.subject320102 Genética clínicaen_US
dc.subject.otherMouse Modelen_US
dc.subject.otherHyperoxaluriaen_US
dc.subject.otherVirus Gene Transferen_US
dc.titlePhenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transferen_US
dc.typeinfo:eu-repo/semantics/articleen_US
dc.typeArticleen_US
dc.identifier.doi10.1038/mt.2010.270en_US
dc.identifier.scopus79955581947-
dc.contributor.authorscopusid14023538500-
dc.contributor.authorscopusid57194487859-
dc.contributor.authorscopusid55617275900-
dc.contributor.authorscopusid14057772100-
dc.contributor.authorscopusid7005798614-
dc.contributor.authorscopusid56712278500-
dc.description.lastpage875en_US
dc.description.firstpage870en_US
dc.relation.volume19en_US
dc.investigacionCiencias de la Saluden_US
dc.type2Artículoen_US
dc.description.numberofpages6en_US
dc.utils.revisionen_US
dc.date.coverdateMayo 2011en_US
dc.identifier.ulpgcen_US
dc.contributor.buulpgcBU-MEDen_US
dc.description.sjr3,425
dc.description.jcr6,873
dc.description.sjrqQ1
dc.description.jcrqQ1
dc.description.scieSCIE
item.fulltextSin texto completo-
item.grantfulltextnone-
crisitem.author.deptGIR IUIBS: Rendimiento humano, ejercicio físico y salud-
crisitem.author.deptIU de Investigaciones Biomédicas y Sanitarias-
crisitem.author.deptDepartamento de Ciencias Clínicas-
crisitem.author.orcid000-0002-1075-9948-
crisitem.author.parentorgIU de Investigaciones Biomédicas y Sanitarias-
crisitem.author.fullNameSantana Rodríguez, Alfredo-
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