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Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria.
Córdoba, Karol M; Serrano-Mendioroz, Irantzu; Jericó, Daniel; Merino, María; Jiang, Lei; Sampedro, Ana; Alegre, Manuel; Corrales, Fernando; Garrido, María J; Martini, Paolo G V; Lanciego, José Luis; Prieto, Jesús; Berraondo, Pedro; Fontanellas, Antonio.
Afiliación
  • Córdoba KM; Hepatology Program, Center for Applied Medical Research (CIMA), University of Navarra, 31008 Pamplona, Spain.
  • Serrano-Mendioroz I; Navarra Institute for Health Research (IDISNA), 31008 Pamplona, Spain.
  • Jericó D; Hepatology Program, Center for Applied Medical Research (CIMA), University of Navarra, 31008 Pamplona, Spain.
  • Merino M; Navarra Institute for Health Research (IDISNA), 31008 Pamplona, Spain.
  • Jiang L; Hepatology Program, Center for Applied Medical Research (CIMA), University of Navarra, 31008 Pamplona, Spain.
  • Sampedro A; Navarra Institute for Health Research (IDISNA), 31008 Pamplona, Spain.
  • Alegre M; Department of Pharmacy and Pharmaceutical Technology, School of Pharmacy, University of Navarra, 31008 Pamplona, Spain.
  • Corrales F; Moderna Inc., Cambridge, MA 02139, USA.
  • Garrido MJ; Hepatology Program, Center for Applied Medical Research (CIMA), University of Navarra, 31008 Pamplona, Spain.
  • Martini PGV; Navarra Institute for Health Research (IDISNA), 31008 Pamplona, Spain.
  • Lanciego JL; Neurophysiology Laboratory, Neuroscience Area, CIMA and Clínica Universitaria, University of Navarra, 31008 Pamplona, Spain.
  • Prieto J; Proteomics Unit, Centro Nacional de Biotecnología (CSIC), 28049 Madrid, Spain.
  • Berraondo P; Department of Pharmacy and Pharmaceutical Technology, School of Pharmacy, University of Navarra, 31008 Pamplona, Spain.
  • Fontanellas A; Moderna Inc., Cambridge, MA 02139, USA.
Sci Transl Med ; 14(627): eabc0700, 2022 01 12.
Article en En | MEDLINE | ID: mdl-35020410
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Hidroximetilbilano Sintasa / Porfiria Intermitente Aguda Límite: Animals Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2022 Tipo del documento: Article País de afiliación: España
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Hidroximetilbilano Sintasa / Porfiria Intermitente Aguda Límite: Animals Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2022 Tipo del documento: Article País de afiliación: España