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

• Main Authors: Daniel Jericó (Author), Karol M. Córdoba (Author), Lei Jiang (Author), Caroline Schmitt (Author), María Morán (Author), Ana Sampedro (Author), Manuel Alegre (Author), María Collantes (Author), Eva Santamaría (Author), Estíbaliz Alegre (Author), Corinne Culerier (Author), Ander Estella-Hermoso de Mendoza (Author), Julen Oyarzabal (Author), Miguel A. Martín (Author), Iván Peñuelas (Author), Matías A. Ávila (Author), Laurent Gouya (Author), Paolo G.V. Martini (Author), Antonio Fontanellas (Author)
• Format: Book
• Published: Elsevier, 2021-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...