Publicaciones en las que colabora con Pedro Berraondo López (10)

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

2019

  1. Messenger RNA therapy for rare genetic metabolic diseases

    Gut, Vol. 68, Núm. 7, pp. 1323-1330

2018

  1. An Inducible Promoter Responsive to Different Porphyrinogenic Stimuli Improves Gene Therapy Vectors for Acute Intermittent Porphyria

    Human Gene Therapy, Vol. 29, Núm. 4, pp. 480-491

  2. 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

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

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

2016

  1. Emerging therapies for acute intermittent porphyria

    Expert Reviews in Molecular Medicine, Vol. 18

2009

  1. Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders

    Human Gene Therapy, Vol. 20, Núm. 8, pp. 908-917