Loss of transcriptional control over endogenous retroelements during reprogramming to pluripotency M Friedli, P Turelli, A Kapopoulou, B Rauwel, N Castro-Díaz, HM Rowe, ... Genome research 24 (8), 1251-1259, 2014 | 120 | 2014 |
Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice C Unzu, A Sampedro, I Mauleón, M Alegre, SG Beattie, ... Molecular Therapy 19 (2), 243-250, 2011 | 81 | 2011 |
Safety and Liver Transduction Efficacy of rAAV5-cohPBGD in Nonhuman Primates: A Potential Therapy for Acute Intermittent Porphyria A Paneda, E Lopez-Franco, C Kaeppel, C Unzu, AG Gil-Royo, D D'Avola, ... Human gene therapy 24 (12), 1007-1017, 2013 | 72 | 2013 |
Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates C Unzu, S Hervás-Stubbs, A Sampedro, I Mauleón, U Mancheño, C Alfaro, ... Journal of translational medicine 10, 1-11, 2012 | 72 | 2012 |
Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent … C Unzu, A Sampedro, I Mauleón, L Vanrell, J Dubrot, RE de Salamanca, ... Journal of hepatology 52 (3), 417-424, 2010 | 38 | 2010 |
Intensive pharmacological immunosuppression allows for repetitive liver gene transfer with recombinant adenovirus in nonhuman primates A Fontanellas, S Hervás-Stubbs, I Mauleón, J Dubrot, U Mancheño, ... Molecular Therapy 18 (4), 754-765, 2010 | 35 | 2010 |
Gene therapy for liver diseases—progress and challenges N Zabaleta, C Unzu, ND Weber, G Gonzalez-Aseguinolaza Nature Reviews Gastroenterology & Hepatology 20 (5), 288-305, 2023 | 32 | 2023 |
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography M Florea, F Nicolaou, S Pacouret, EM Zinn, J Sanmiguel, ... Molecular Therapy Methods & Clinical Development 28, 146-159, 2023 | 31 | 2023 |
Pharmacological induction of a progenitor state for the efficient expansion of primary human hepatocytes C Unzu, E Planet, N Brandenberg, F Fusil, M Cassano, J Perez‐Vargas, ... Hepatology 69 (5), 2214-2231, 2019 | 30 | 2019 |
Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate E Andres-Mateos, LD Landegger, C Unzu, J Phillips, BM Lin, NA Dewyer, ... Nature Communications 13 (1), 1359, 2022 | 25 | 2022 |
Helper-dependent adenoviral liver gene therapy protects against induced attacks and corrects protein folding stress in acute intermittent porphyria mice C Unzu, A Sampedro, I Mauleon, M Gonzalez-Aparicio, ... Human molecular genetics 22 (14), 2929-2940, 2013 | 25 | 2013 |
Renal failure affects the enzymatic activities of the three first steps in hepatic heme biosynthesis in the acute intermittent porphyria mouse C Unzu, A Sampedro, E Sardh, I Mauleón, R Enríquez de Salamanca, ... PLoS One 7 (3), e32978, 2012 | 20 | 2012 |
Ancestral library identifies conserved reprogrammable liver motif on AAV capsid E Zinn, C Unzu, PF Schmit, HT Turunen, N Zabaleta, J Sanmiguel, ... Cell Reports Medicine 3 (11), 2022 | 18 | 2022 |
Helper-dependent adenovirus achieve more efficient and persistent liver transgene expression in non-human primates under immunosuppression C Unzu, I Melero, S Hervás-Stubbs, A Sampedro, U Mancheño, ... Gene therapy 22 (11), 856-865, 2015 | 17 | 2015 |
Innate functions of immunoglobulin M lessen liver gene transfer with helper-dependent adenovirus C Unzu, I Melero, A Morales-Kastresana, A Sampedro, ... PLoS One 9 (1), e85432, 2014 | 10 | 2014 |
Understanding and tackling immune responses to adeno-associated viral vectors H Costa-Verdera, C Unzu, E Valeri, S Adriouch, ... Human Gene Therapy 34 (17-18), 836-852, 2023 | 7 | 2023 |
Human Hepatocyte‐Derived Induced Pluripotent Stem Cells: MYC Expression, Similarities to Human Germ Cell Tumors, and Safety Issues C Unzu, M Friedli, A Bosman, ME Jaconi, BE Wildhaber, AL Rougemont Stem cells international 2016 (1), 4370142, 2016 | 6 | 2016 |
Epidermal growth factor receptor ligands in murine models for erythropoietic protoporphyria: potential novel players in the progression of liver injury C Berasain, A Sampedro, I Mauleon, S Goñi, MU Latasa, N Matscheko, ... Cellular and Molecular Biology 55 (1), 29-37, 2009 | 5 | 2009 |
Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation M Cabanes-Creus, SHY Liao, R Gale Navarro, M Knight, D Nazareth, ... Nature Communications 15 (1), 1876, 2024 | 2 | 2024 |
Optimized surgical approach leads to highly efficient AAV gene transfer to inner hair cells in rhesus macaque E Andres-Mateos, LD Landegger, C Unzu, J Phillips, B Lin, NA Dewyer, ... MOLECULAR THERAPY 27 (4), 318-318, 2019 | 2 | 2019 |