Publications en collaboration avec des chercheurs de Centro de Investigación Biomédica en Red sobre Enfermedades Hepáticas y Digestivas (16)

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

2023

  1. Nutritional Interventions with Bacillus coagulans Improved Glucose Metabolism and Hyperinsulinemia in Mice with Acute Intermittent Porphyria

    International Journal of Molecular Sciences, Vol. 24, Núm. 15

2022

  1. Messenger RNA as a personalized therapy: The moment of truth for rare metabolic diseases

    International Review of Cell and Molecular Biology (Elsevier Inc.), pp. 55-96

  2. Recent Insights into the Pathogenesis of Acute Porphyria Attacks and Increasing Hepatic PBGD as an Etiological Treatment

    Life, Vol. 12, Núm. 11

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

    Science Translational Medicine, Vol. 14, Núm. 627

2021

  1. High prevalence of insulin resistance in asymptomatic patients with acute intermittent porphyria and liver-targeted insulin as a novel therapeutic approach

    Biomedicines, Vol. 9, Núm. 3, pp. 1-18

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

    Molecular Therapy - Nucleic Acids, Vol. 25, pp. 207-219

2020

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

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

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

  2. Non-parenchymal TREM-2 protects the liver from immune-mediated hepatocellular damage

    Gut, Vol. 68, Núm. 3, pp. 533-546

2018

  1. Bioengineered PBGD variant improves the therapeutic index of gene therapy vectors for acute intermittent porphyria

    Human Molecular Genetics, Vol. 27, Núm. 21, pp. 3688-3696

  2. Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

    Nature Medicine, Vol. 24, Núm. 12, pp. 1899-1909

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

2011

  1. Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice

    Molecular Therapy, Vol. 19, Núm. 2, pp. 243-250

2010

  1. Intensive pharmacological immunosuppression allows for repetitive liver gene transfer with recombinant adenovirus in nonhuman primates

    Molecular Therapy, Vol. 18, Núm. 4, pp. 754-765

2009

  1. PET imaging of thymidine kinase gene expression in the liver of non-human primates following systemic delivery of an adenoviral vector

    Gene Therapy, Vol. 16, Núm. 1, pp. 136-141