mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks

Variegate porphyria (VP) results from haploinsufficiency of protoporphyrinogen oxidase (PPOX), the seventh enzyme in the heme synthesis pathway. There is no VP model that recapitulates the clinical manifestations of acute attacks. Combined administrations of 2-allyl-2-isopropylacetamide and rifampic...

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Detalles Bibliográficos
Autores principales: Jericó, Daniel, Córdoba, Karol M., Jiang, Lei, Schmitt, Caroline, Morán, María, Sampedro, Ana, Alegre, Manuel, Collantes, María, Santamaría, Eva, Alegre, Estíbaliz, Culerier, Corinne, de Mendoza, Ander Estella-Hermoso, Oyarzabal, Julen, Martín, Miguel A., Peñuelas, Iván, Ávila, Matías A., Gouya, Laurent, Martini, Paolo G.V., Fontanellas, Antonio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8368795/
https://www.ncbi.nlm.nih.gov/pubmed/34458006
http://dx.doi.org/10.1016/j.omtn.2021.05.010

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