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| Highly efficient therapeutic gene editing of human hematopoietic stem cells Y Wu, J Zeng, BP Roscoe, P Liu, Q Yao, CR Lazzarotto, K Clement, ... Nature medicine 25 (5), 776-783, 2019 | 498 | 2019 |
| An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities JM Gehrke, O Cervantes, MK Clement, Y Wu, J Zeng, DE Bauer, L Pinello, ... Nature biotechnology 36 (10), 977-982, 2018 | 478 | 2018 |
| Therapeutic base editing of human hematopoietic stem cells J Zeng, Y Wu, C Ren, J Bonanno, AH Shen, D Shea, JM Gehrke, ... Nature Medicine 26 (4), 535-541, 2020 | 274 | 2020 |
| Genome editing of HBG1 and HBG2 to induce fetal hemoglobin JY Métais, PA Doerfler, T Mayuranathan, DE Bauer, SC Fowler, MM Hsieh, ... Blood advances 3 (21), 3379-3392, 2019 | 165 | 2019 |
| Variant-aware saturating mutagenesis using multiple Cas9 nucleases identifies regulatory elements at trait-associated loci MC Canver, S Lessard, L Pinello, Y Wu, Y Ilboudo, EN Stern, ... Nature genetics 49 (4), 625-634, 2017 | 121 | 2017 |
| Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia S Xu, K Luk, Q Yao, AH Shen, J Zeng, Y Wu, HY Luo, C Brendel, L Pinello, ... Blood, The Journal of the American Society of Hematology 133 (21), 2255-2262, 2019 | 88 | 2019 |
| Small-molecule PAPD5 inhibitors restore telomerase activity in patient stem cells N Nagpal, J Wang, J Zeng, E Lo, DH Moon, K Luk, RO Braun, ... Cell Stem Cell 26 (6), 896-909. e8, 2020 | 86 | 2020 |
| Human genetic diversity alters off-target outcomes of therapeutic gene editing S Cancellieri, J Zeng, LY Lin, M Tognon, MA Nguyen, J Lin, N Bombieri, ... Nature genetics 55 (1), 34-43, 2023 | 78 | 2023 |
| BCL11A enhancer–edited hematopoietic stem cells persist in rhesus monkeys without toxicity S Demirci, J Zeng, Y Wu, N Uchida, AH Shen, D Pellin, J Gamer, ... The Journal of clinical investigation 130 (12), 6677-6687, 2020 | 68 | 2020 |
| ZNF410 represses fetal globin by singular control of CHD4 DS Vinjamur, Q Yao, MA Cole, C McGuckin, C Ren, J Zeng, M Hossain, ... Nature genetics 53 (5), 719-728, 2021 | 56 | 2021 |
| Epitope editing enables targeted immunotherapy of acute myeloid leukaemia G Casirati, A Cosentino, A Mucci, M Salah Mahmoud, I Ugarte Zabala, ... Nature 621 (7978), 404-414, 2023 | 53 | 2023 |
| Dissecting ELANE neutropenia pathogenicity by human HSC gene editing S Rao, Y Yao, JS de Brito, Q Yao, AH Shen, RE Watkinson, AL Kennedy, ... Cell Stem Cell 28 (5), 833-845. e5, 2021 | 38 | 2021 |
| Optimization of nuclear localization signal composition improves CRISPR-cas12a editing rates in human primary cells K Luk, P Liu, J Zeng, Y Wang, SA Maitland, F Idrizi, K Ponnienselvan, ... GEN biotechnology 1 (3), 271-284, 2022 | 18 | 2022 |
| Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells J Zeng, MA Nguyen, P Liu, LF Da Silva, S Levesque, LY Lin, DG Justus, ... Cell Stem Cell, 2023 | 11 | 2023 |
| Durable and robust fetal globin induction without Anemia in rhesus monkeys following autologous hematopoietic stem cell transplant with BCL11A Erythroid enhancer editing S Demirci, J Zeng, Y Wu, N Uchida, J Gamer, M Yapundich, C Drysdale, ... Blood 134, 4632, 2019 | 5 | 2019 |
| Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation K Essawi, W Hakami, MBN Khan, R Martin, J Zeng, R Chu, N Uchida, ... Molecular Therapy Methods & Clinical Development 29, 483-493, 2023 | 4 | 2023 |
| Gene editing ELANE in human hematopoietic stem and progenitor cells reveals disease mechanisms and therapeutic strategies for severe congenital neutropenia S Rao, J Brito-Frazao, AV Serbin, Q Yao, K Luk, Y Wu, J Zeng, C Ren, ... Blood 134, 3, 2019 | 4 | 2019 |
| Combined+ 58 and+ 55 BCL11A enhancer editing Yields exceptional efficiency, specificity and HbF induction in human and NHP preclinical models J Zeng, S Demirci, MA Nguyen, LY Lin, SA Maitland, E Mintzer, Y Wu, ... Blood 138, 1852, 2021 | 3 | 2021 |
| Enhanced bcl11a rnp/crispr delivery & editing using a 3xnls-cas9 DE Bauer, SA Wolfe, LIU Pengpeng, WU Yuxuan, J Zeng | 3 | 2021 |
