Publicaciones en colaboración con investigadores/as de Centro de Investigación Biomédica en Red sobre Enfermedades Hepáticas y Digestivas (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

2023

  1. Engineering U1-Based Tetracycline-Inducible Riboswitches to Control Gene Expression in Mammals

    ACS Nano, Vol. 17, Núm. 23, pp. 23331-23346

2021

  1. Gene supplementation of CYP27A1 in the liver restores bile acid metabolism in a mouse model of cerebrotendinous xanthomatosis

    Molecular Therapy - Methods and Clinical Development, Vol. 22, pp. 210-221

2018

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

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

2017

  1. X-box Binding Protein 1 Regulates Unfolded Protein, Acute-Phase, and DNA Damage Responses During Regeneration of Mouse Liver

    Gastroenterology, Vol. 152, Núm. 5, pp. 1203-1216.e15

2016

  1. Capsid-deficient alphaviruses generate propagative infectious microvesicles at the plasma membrane

    Cellular and Molecular Life Sciences, Vol. 73, Núm. 20, pp. 3897-3916

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