| Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells G Santilli, E Almarza, C Brendel, U Choi, C Beilin, MP Blundell, S Haria, ... Molecular Therapy 19 (1), 122-132, 2011 | 166 | 2011 |
| PARP‐2 deficiency affects the survival of CD4+ CD8+ double‐positive thymocytes J Yélamos, Y Monreal, L Saenz, E Aguado, V Schreiber, R Mota, T Fuente, ... The EMBO journal 25 (18), 4350-4360, 2006 | 155 | 2006 |
| Leukocyte adhesion deficiency-I: A comprehensive review of all published cases EA Novoa, S Kasbekar, AJ Thrasher, DB Kohn, J Sevilla, T Nguyen, ... The Journal of Allergy and Clinical Immunology: In Practice 6 (4), 1418-1420 …, 2018 | 112 | 2018 |
| Targeted gene therapy and cell reprogramming in F anconi anemia P Rio, R Baños, A Lombardo, O Quintana‐Bustamante, L Alvarez, ... EMBO molecular medicine 6 (6), 835-848, 2014 | 97 | 2014 |
| Efficient non-viral gene delivery into human hematopoietic stem cells by minicircle sleeping beauty transposon vectors M Holstein, C Mesa-Nuñez, C Miskey, E Almarza, V Poletti, M Schmeer, ... Molecular therapy 26 (4), 1137-1153, 2018 | 77 | 2018 |
| The application of nanoparticles in gene therapy and magnetic resonance imaging F Herranz, E Almarza, I Rodríguez, B Salinas, Y Rosell, M Desco, ... Microscopy research and technique 74 (7), 577-591, 2011 | 68 | 2011 |
| Development of lentiviral vectors with optimized transcriptional activity for the gene therapy of patients with Fanconi anemia A Gonzalez-Murillo, ML Lozano, L Alvarez, A Jacome, E Almarza, ... Human gene therapy 21 (5), 623-630, 2010 | 66 | 2010 |
| In vivo imaging of lung inflammation with neutrophil-specific 68Ga nano-radiotracer J Pellico, AV Lechuga-Vieco, E Almarza, A Hidalgo, C Mesa-Nuñez, ... Scientific reports 7 (1), 13242, 2017 | 58 | 2017 |
| Parallel multifunctionalization of nanoparticles: a one-step modular approach for in vivo imaging H Groult, J Ruiz-Cabello, J Pellico, AV Lechuga-Vieco, R Bhavesh, ... Bioconjugate chemistry 26 (1), 153-160, 2015 | 48 | 2015 |
| Characteristics of lentiviral vectors harboring the proximal promoter of the vav proto-oncogene: a weak and efficient promoter for gene therapy E Almarza, P Río, NW Meza, M Aldea, X Agirre, G Guenechea, ... Molecular Therapy 15 (8), 1487-1494, 2007 | 40 | 2007 |
| Regulatory elements of the vav gene drive transgene expression in hematopoietic stem cells from adult mice E Almarza, JC Segovia, G Guenechea, SG Gómez, Á Ramı́rez, JA Bueren Experimental hematology 32 (4), 360-364, 2004 | 35 | 2004 |
| Advances in the gene therapy of monogenic blood cell diseases JA Bueren, O Quintana‐Bustamante, E Almarza, S Navarro, P Río, ... Clinical genetics 97 (1), 89-102, 2020 | 31 | 2020 |
| Lentiviral vector-mediated correction of a mouse model of leukocyte adhesion deficiency type I D Leon-Rico, M Aldea, R Sanchez-Baltasar, C Mesa-Nunez, J Record, ... Human gene therapy 27 (9), 668-678, 2016 | 27 | 2016 |
| Generation of iPSCs from Genetically Corrected Brca2 Hypomorphic Cells: Implications in Cell Reprogramming and Stem Cell Therapy S Navarro, V Moleiro, FJ Molina-Estevez, ML Lozano, R Chinchon, ... Stem Cells 32 (2), 436-446, 2014 | 20 | 2014 |
| Comparison of haematopoietic stem cell engraftment through the retro-orbital venous sinus and the lateral vein: alternative routes for bone marrow transplantation in mice D Leon-Rico, M Fernández-García, M Aldea, R Sánchez, M Peces-Barba, ... Laboratory Animals 49 (2), 132-141, 2015 | 18 | 2015 |
| Correction of SCID-X1 Using an Enhancerless Vav Promoter E Almarza, F Zhang, G Santilli, MP Blundell, SJ Howe, SI Thornhill, ... Human gene therapy 22 (3), 263-270, 2011 | 18 | 2011 |
| A phase 1/2 study of lentiviral-mediated ex-vivo gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I): results from phase 1 DB Kohn, GR Rao, E Almarza, D Terrazas, E Nicoletti, A Fernandes, ... Blood 136, 15, 2020 | 15 | 2020 |
| Brief report: reduced expression of CD18 leads to the in vivo expansion of hematopoietic stem cells in mouse bone marrow D Leon-Rico, M Aldea, R Sanchez, JC Segovia, LA Weiss, A Hidalgo, ... Stem Cells 32 (10), 2794-2798, 2014 | 13 | 2014 |
| Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal I Moscatelli, E Almarza, A Schambach, D Ricks, A Schulz, CD Herzog, ... Molecular Therapy Methods & Clinical Development 20, 389-397, 2021 | 11 | 2021 |
| Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I C Mesa-Núñez, C Damián, M Fernández-García, B Díez, G Rao, ... Molecular Therapy Methods & Clinical Development 26, 459-470, 2022 | 7 | 2022 |
