Please use this identifier to cite or link to this item:
http://hdl.handle.net/10553/46097
DC Field | Value | Language |
---|---|---|
dc.contributor.author | Salido, Eduardo | en_US |
dc.contributor.author | Rodriguez-Pena, Marisol | en_US |
dc.contributor.author | Santana, Alfredo | en_US |
dc.contributor.author | Beattie, Stuart G. | en_US |
dc.contributor.author | Petry, Harald | en_US |
dc.contributor.author | Torres, Armando | en_US |
dc.date.accessioned | 2018-11-23T01:21:09Z | - |
dc.date.available | 2018-11-23T01:21:09Z | - |
dc.date.issued | 2011 | en_US |
dc.identifier.issn | 1525-0016 | en_US |
dc.identifier.uri | http://hdl.handle.net/10553/46097 | - |
dc.description.abstract | Primary 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.language | eng | en_US |
dc.relation.ispartof | Molecular Therapy | en_US |
dc.source | Molecular Therapy [ISSN 1525-0016],v. 19, p. 870-875 | en_US |
dc.subject | 32 Ciencias médicas | en_US |
dc.subject | 320102 Genética clínica | en_US |
dc.subject.other | Mouse Model | en_US |
dc.subject.other | Hyperoxaluria | en_US |
dc.subject.other | Virus Gene Transfer | en_US |
dc.title | Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer | en_US |
dc.type | info:eu-repo/semantics/article | en_US |
dc.type | Article | en_US |
dc.identifier.doi | 10.1038/mt.2010.270 | en_US |
dc.identifier.scopus | 79955581947 | - |
dc.contributor.authorscopusid | 14023538500 | - |
dc.contributor.authorscopusid | 57194487859 | - |
dc.contributor.authorscopusid | 55617275900 | - |
dc.contributor.authorscopusid | 14057772100 | - |
dc.contributor.authorscopusid | 7005798614 | - |
dc.contributor.authorscopusid | 56712278500 | - |
dc.description.lastpage | 875 | en_US |
dc.description.firstpage | 870 | en_US |
dc.relation.volume | 19 | en_US |
dc.investigacion | Ciencias de la Salud | en_US |
dc.type2 | Artículo | en_US |
dc.description.numberofpages | 6 | en_US |
dc.utils.revision | Sí | en_US |
dc.date.coverdate | Mayo 2011 | en_US |
dc.identifier.ulpgc | Sí | en_US |
dc.contributor.buulpgc | BU-MED | en_US |
dc.description.sjr | 3,425 | |
dc.description.jcr | 6,873 | |
dc.description.sjrq | Q1 | |
dc.description.jcrq | Q1 | |
dc.description.scie | SCIE | |
item.fulltext | Sin texto completo | - |
item.grantfulltext | none | - |
crisitem.author.dept | GIR IUIBS: Rendimiento humano, ejercicio físico y salud | - |
crisitem.author.dept | IU de Investigaciones Biomédicas y Sanitarias | - |
crisitem.author.dept | Departamento de Ciencias Clínicas | - |
crisitem.author.orcid | 000-0002-1075-9948 | - |
crisitem.author.parentorg | IU de Investigaciones Biomédicas y Sanitarias | - |
crisitem.author.fullName | Santana Rodríguez, Alfredo | - |
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