Ana Sampedro Pascual-rekin lankidetzan egindako argitalpenak (8)

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. 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

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