Publicaciones en colaboración con investigadores/as de Clínica Universitaria de Navarra (11)

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

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. 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

  2. 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

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

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

  2. 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