Publicacións en colaboración con investigadores/as de Instituto de Investigación Sanitaria de Navarra (12)

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

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

2018

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

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

2016

  1. Glucose metabolism during fasting is altered in experimental porphobilinogen deaminase deficiency

    Human Molecular Genetics, Vol. 25, Núm. 7, pp. 1318-1327

2015

  1. Helper-dependent adenovirus achieve more efficient and persistent liver transgene expression in non-human primates under immunosuppression

    Gene Therapy, Vol. 22, Núm. 11, pp. 856-865

  2. Vitamin D-binding protein as a biomarker of active disease in acute intermittent porphyria

    Journal of Proteomics, Vol. 127, pp. 377-385