Carmen Unzu Ezquerro-rekin lankidetzan egindako argitalpenak (8)

2024

  1. Enhanced AAV transduction across preclinical CNS models: A comparative study in human brain organoids with cross-species evaluations

    Molecular Therapy Nucleic Acids, Vol. 35, Núm. 3

  2. Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation

    Nature Communications, Vol. 15, Núm. 1

2023

  1. Gene therapy for liver diseases — progress and challenges

    Nature Reviews Gastroenterology and Hepatology, Vol. 20, Núm. 5, pp. 288-305

  2. Topoisomerase Inhibitors Increase Episomal DNA Expression by Inducing the Integration of Episomal DNA in Hepatic Cells

    Pharmaceutics, Vol. 15, Núm. 10

  3. Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors

    Human gene therapy, Vol. 34, Núm. 17-18, pp. 836-852

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

2010

  1. 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