| A comprehensive, ethnically diverse library of sickle cell disease-specific induced pluripotent stem cells S Park, A Gianotti-Sommer, FJ Molina-Estevez, K Vanuytsel, N Skvir, ... Stem Cell Reports 8 (4), 1076-1085, 2017 | 46 | 2017 |
| Modeling APC mutagenesis and familial adenomatous polyposis using human iPS cells CA Sommer, A Capilla, FJ Molina-Estevez, A Gianotti-Sommer, N Skvir, ... PLoS One 13 (7), e0200657, 2018 | 33 | 2018 |
| Lentiviral-mediated gene therapy in Fanconi anemia-A mice reveals long-term engraftment and continuous turnover of corrected HSCs F Javier Molina-Estevez, A Nowrouzi, ML Lozano, A Galy, S Charrier, ... Current gene therapy 15 (6), 550-562, 2015 | 26 | 2015 |
| 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 |
| Brief report: impaired cell reprogramming in nonhomologous end joining deficient cells FJ Molina-Estevez, ML Lozano, S Navarro, Y Torres, I Grabundzija, Z Ivics, ... Stem Cells 31 (8), 1726-1730, 2013 | 18 | 2013 |
| Efficacy and safety of universal (TCRKO) ARI-0001 CAR-T cells for the treatment of B-cell lymphoma N Maldonado-Perez, M Tristán-Manzano, P Justicia-Lirio, ... Frontiers in Immunology 13, 1011858, 2022 | 8 | 2022 |
| Molecular evidence of genome editing in a mouse model of immunodeficiency HH Abdul-Razak, CJ Rocca, SJ Howe, ME Alonso-Ferrero, J Wang, ... Scientific reports 8 (1), 8214, 2018 | 7 | 2018 |
| Lentiviral vectors for inducible, transactivator-free advanced therapy medicinal products: Application to CAR-T cells M Tristán-Manzano, N Maldonado-Pérez, P Justicia-Lirio, ... Molecular Therapy-Nucleic Acids 32, 322-339, 2023 | 1 | 2023 |
| Isogenic GAA-KO murine muscle cell lines mimicking severe Pompe mutations as preclinical models for the screening of potential gene therapy strategies A Aguilar-González, JE González-Correa, E Barriocanal-Casado, ... International Journal of Molecular Sciences 23 (11), 6298, 2022 | 1 | 2022 |
| Gene therapy and cell reprogramming in mouse models of monogenic hematopoietic stem cell diseases GG Amurrio Universidad Autónoma de Madrid, 2013 | 1 | 2013 |
| First-in-class Transactivator-Free, Doxycycline-inducible IL-18-engineered CAR-T cells for relapsed/refractory B-cell lymphomas PL Justicia-Lirio, M Tristan-Manzano, N Maldonado-Perez, ... bioRxiv, 2024.01. 23.576842, 2024 | | 2024 |
| Generation of first-in-class doxycycline-inducible IL-18 releasing CAR-T cells targeting solid tumors P Justicia-Lirio, M Tristan-Manzano, N Maldonado-Perez, ... HUMAN GENE THERAPY 33 (23-24), A14-A14, 2022 | | 2022 |
| A negative regulatory region in WAS intron 1 controls megakaryocytic differentiation A Hinckley Boned, S Sanchez Hernandez, A Aguilar Gonzalez, ... HUMAN GENE THERAPY 33 (23-24), A22-A22, 2022 | | 2022 |
| CX3CR1 intron 4 targeting provides a novel safe harbour for Trojan Horse strategies to treat monogenic disorders I Ramos-Hernandez, N Maldonado-Perez, P Munoz, FJ Molina-Estevez, ... HUMAN GENE THERAPY 33 (23-24), A148-A148, 2022 | | 2022 |
| Modeling rare monogenic disorders as a translational tool: CRISPR/CAS9 editing of Pompe Mutations I Ramos-Hernandez, A Aguilar-Gonzalez, E Barriocanal, P Munoz, ... HUMAN GENE THERAPY 32 (19-20), A70-A70, 2021 | | 2021 |
| Generation of Pompe disease cellular models to analyze gene therapy aproaches using optimized GAA A Aguilar-Gonzalez, JE Gonzalez-Correa, E Barriocanal-Casado, ... HUMAN GENE THERAPY 32 (19-20), A124-A125, 2021 | | 2021 |
| Optimizing genome editing approaches for safe transgene expression in monogenic disorders I Ramos-Hernandez, FJ Molina-Estevez, P Munoz, F Martin HUMAN GENE THERAPY 32 (19-20), A70-A71, 2021 | | 2021 |
| Molecular Evidence of Ex Vivo Gene Editing in a Mouse Model of Immunodeficiency HH Abdul-Razak, C Rocca, SJ Howe, ME Alonso-Ferrero, J Wang, ... MOLECULAR THERAPY 25 (5), 303-303, 2017 | | 2017 |
| Modeling Zaat-Driven Proteotoxicity Using Crispr-Edited Syngeneic Patient-Ipscs J Kaserman, FJ Molina Estevez, M Higgins, G Mostoslavsky, DN Kotton, ... D108. CELL FATE DECISIONS: SENESCENCE, REPAIR, AND REGENERATION, A7495-A7495, 2017 | | 2017 |
| Crispr-Directed Editing Of Patient-Ipscs To Determine Genetic Contributors To Zaat-Driven Proteotoxicity J Kaserman, FJ Molina Estevez, GC Lonza, G Mostoslavsky, DN Kotton, ... B28. ADVANCES IN STEM CELLS IN INJURY AND REPAIR FOR 2016, A3093-A3093, 2016 | | 2016 |
