Publications by the researcher in collaboration with Carmen Unzu Ezquerro (7)

2013

  1. Helper-dependent adenoviral liver gene therapy protects against induced attacks and corrects protein folding stress in acute intermittent porphyria mice

    Human Molecular Genetics, Vol. 22, Núm. 14, pp. 2929-2940

  2. 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

2012

  1. Renal failure affects the enzymatic activities of the three first steps in hepatic heme biosynthesis in the acute intermittent porphyria mouse

    PLoS ONE, Vol. 7, Núm. 3

  2. Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates.

    Journal of translational medicine, Vol. 10, pp. 122

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

2010

  1. Intensive pharmacological immunosuppression allows for repetitive liver gene transfer with recombinant adenovirus in nonhuman primates

    Molecular Therapy, Vol. 18, Núm. 4, pp. 754-765

  2. Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent porphyria

    Journal of Hepatology, Vol. 52, Núm. 3, pp. 417-424