Publicaciones (21) Publicaciones de Carmen Unzu Ezquerro

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. High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography

    Molecular Therapy - Methods and Clinical Development, Vol. 28, pp. 146-159

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

    Pharmaceutics, Vol. 15, Núm. 10

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

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

2022

  1. Ancestral library identifies conserved reprogrammable liver motif on AAV capsid

    Cell Reports Medicine, Vol. 3, Núm. 11

  2. Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate

    Nature Communications, Vol. 13, Núm. 1

2019

  1. Pharmacological Induction of a Progenitor State for the Efficient Expansion of Primary Human Hepatocytes

    Hepatology, Vol. 69, Núm. 5, pp. 2214-2231

2016

  1. Human hepatocyte-derived induced pluripotent stem cells: MYC expression, similarities to human germ cell tumors, and safety issues

    Stem Cells International, Vol. 2016

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

2014

  1. Innate functions of immunoglobulin M lessen liver gene transfer with helper-dependent adenovirus

    PLoS ONE, Vol. 9, Núm. 1

  2. Loss of transcriptional control over endogenous retroelements during reprogramming to pluripotency

    Genome Research, Vol. 24, Núm. 8, pp. 1251-1259

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