Publicaciones en las que colabora con Antonio Fontanellas Roma (7)

2024

  1. Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates

    Gut

2022

  1. Mechanistic modelling of enzyme-restoration effects of new recombinant liver-targeted proteins in acute intermittent porphyria

    British Journal of Pharmacology, Vol. 179, Núm. 14, pp. 3815-3830

  2. Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria

    Science Translational Medicine, Vol. 14, Núm. 627

2020

  1. Brain ventricular enlargement in human and murine acute intermittent porphyria

    Human molecular genetics, Vol. 29, Núm. 19, pp. 3211-3223

  2. Disease pharmacokinetic–pharmacodynamic modelling in acute intermittent porphyria to support the development of mRNA-based therapies

    British Journal of Pharmacology, Vol. 177, Núm. 14, pp. 3168-3182

2019

  1. Computational disease model of phenobarbital-induced acute attacks in an acute intermittent porphyria mouse model

    Molecular Genetics and Metabolism, Vol. 128, Núm. 3, pp. 367-375

2013

  1. Safety and liver transduction efficacy of raav5-cohpbgd in nonhuman primates: A potential therapy for acute intermittent porphyria

    Human Gene Therapy, Vol. 24, Núm. 12, pp. 1007-1017