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

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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Bibliographic Details
Main Authors: Daniel Jericó, Karol M. Córdoba, Lei Jiang, Caroline Schmitt, María Morán, Ana Sampedro, Manuel Alegre, María Collantes, Eva Santamaría, Estíbaliz Alegre, Corinne Culerier, Ander Estella-Hermoso de Mendoza, Julen Oyarzabal, Miguel A. Martín, Iván Peñuelas, Matías A. Ávila, Laurent Gouya, Paolo G.V. Martini, Antonio Fontanellas
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
mRNA delivery
lipid nanoparticles
rare metabolic disease
hepatic heme synthesis
pharmacological model of variegate porphyria
characterization of variegate porphyria rabbits
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121001268

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http://www.sciencedirect.com/science/article/pii/S2162253121001268

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