| Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells Á Raya, I Rodríguez-Pizà, G Guenechea, R Vassena, S Navarro, ... Nature 460 (7251), 53-59, 2009 | 966 | 2009 |
| The BRCA1-interacting helicase BRIP1 is deficient in Fanconi anemia O Levran, C Attwooll, RT Henry, KL Milton, K Neveling, P Rio, SD Batish, ... Nature genetics 37 (9), 931-933, 2005 | 452 | 2005 |
| Mutations in ERCC4, encoding the DNA-repair endonuclease XPF, cause Fanconi anemia M Bogliolo, B Schuster, C Stoepker, B Derkunt, Y Su, A Raams, JP Trujillo, ... The American Journal of Human Genetics 92 (5), 800-806, 2013 | 378 | 2013 |
| Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia P Rio, S Navarro, W Wang, R Sanchez-Dominguez, RM Pujol, ... Nature medicine 25 (9), 1396-1401, 2019 | 145 | 2019 |
| The transforming acidic coiled coil 3 protein is essential for spindle-dependent chromosome alignment and mitotic survival L Schneider, F Essmann, A Kletke, P Rio, H Hanenberg, W Wetzel, ... Journal of Biological Chemistry 282 (40), 29273-29283, 2007 | 105 | 2007 |
| NKG2D-CAR-transduced natural killer cells efficiently target multiple myeloma A Leivas, A Valeri, L Córdoba, A García-Ortiz, A Ortiz, L Sánchez-Vega, ... Blood Cancer Journal 11 (8), 146, 2021 | 97 | 2021 |
| 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 | 92 | 2014 |
| A comprehensive strategy for the subtyping of patients with Fanconi anaemia: conclusions from the Spanish Fanconi Anemia Research Network JA Casado, E Callén, A Jacome, P Río, M Castella, S Lobitz, T Ferro, ... Journal of medical genetics 44 (4), 241-249, 2007 | 82 | 2007 |
| NHEJ-mediated repair of CRISPR-Cas9-induced DNA breaks efficiently corrects mutations in HSPCs from patients with fanconi anemia FJ Román-Rodríguez, L Ugalde, L Álvarez, B Díez, MJ Ramírez, ... Cell Stem Cell 25 (5), 607-621. e7, 2019 | 78 | 2019 |
| Lentiviral-mediated genetic correction of hematopoietic and mesenchymal progenitor cells from Fanconi anemia patients A Jacome, S Navarro, P Río, RM Yañez, A González-Murillo, ML Lozano, ... Molecular Therapy 17 (6), 1083-1092, 2009 | 73 | 2009 |
| 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 | 63 | 2010 |
| Deficiency of Bruton's tyrosine kinase in B cell precursor leukemia cells N Feldhahn, P Río, BNB Soh, S Liedtke, M Sprangers, F Klein, P Wernet, ... Proceedings of the National Academy of Sciences 102 (37), 13266-13271, 2005 | 63 | 2005 |
| Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients B Diez, P Genovese, FJ Roman‐Rodriguez, L Alvarez, G Schiroli, ... EMBO molecular medicine 9 (11), 1574-1588, 2017 | 62 | 2017 |
| Relevance of the Fanconi anemia pathway in the response of human cells to trabectedin JA Casado, P Rio, E Marco, V Garcia-Hernandez, A Domingo, L Perez, ... Molecular cancer therapeutics 7 (5), 1309-1318, 2008 | 62 | 2008 |
| Gene therapy for Fanconi anemia: one step closer to the clinic J Tolar, PS Becker, DW Clapp, H Hanenberg, CD De Heredia, HP Kiem, ... Human gene therapy 23 (2), 141-144, 2012 | 59 | 2012 |
| Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients P Río, S Navarro, G Guenechea, R Sánchez-Domínguez, ML Lamana, ... Blood, The Journal of the American Society of Hematology 130 (13), 1535-1542, 2017 | 57 | 2017 |
| Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting J Tolar, JE Adair, M Antoniou, CC Bartholomae, PS Becker, BR Blazar, ... Molecular Therapy 19 (7), 1193-1198, 2011 | 57 | 2011 |
| Preclinical activity of LBH589 alone or in combination with chemotherapy in a xenogeneic mouse model of human acute lymphoblastic leukemia A Vilas-Zornoza, X Agirre, G Abizanda, C Moreno, V Segura, ... Leukemia 26 (7), 1517-1526, 2012 | 54 | 2012 |
| A novel lentiviral vector targets gene transfer into human hematopoietic stem cells in marrow from patients with bone marrow failure syndrome and in vivo in humanized mice C Frecha, C Costa, D Negre, F Amirache, D Trono, P Rio, J Bueren, ... Blood, The Journal of the American Society of Hematology 119 (5), 1139-1150, 2012 | 54 | 2012 |
| TACC3 depletion sensitizes to paclitaxel-induced cell death and overrides p21WAF-mediated cell cycle arrest L Schneider, F Essmann, A Kletke, P Rio, H Hanenberg, ... Oncogene 27 (1), 116-125, 2008 | 53 | 2008 |
