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 | 433 | 2019 |
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 | 229 | 2020 |
Evaluating and enhancing target specificity of gene-editing nucleases and deaminases D Kim, K Luk, SA Wolfe, JS Kim Annual review of biochemistry 88, 191-220, 2019 | 138 | 2019 |
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 | 135 | 2019 |
Precise therapeutic gene correction by a simple nuclease-induced double-stranded break S Iyer, S Suresh, D Guo, K Daman, JCJ Chen, P Liu, M Zieger, K Luk, ... Nature 568 (7753), 561-565, 2019 | 109 | 2019 |
Enhanced Cas12a editing in mammalian cells and zebrafish P Liu, K Luk, M Shin, F Idrizi, S Kwok, B Roscoe, E Mintzer, S Suresh, ... Nucleic acids research 47 (8), 4169-4180, 2019 | 100 | 2019 |
Rational targeting of a NuRD subcomplex guided by comprehensive in situ mutagenesis F Sher, M Hossain, D Seruggia, VAC Schoonenberg, Q Yao, P Cifani, ... Nature genetics 51 (7), 1149-1159, 2019 | 92 | 2019 |
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 | 75 | 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 | 72 | 2020 |
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 | 56 | 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 | 46 | 2021 |
CRISPR-enhanced human adipocyte browning as cell therapy for metabolic disease E Tsagkaraki, SM Nicoloro, T DeSouza, J Solivan-Rivera, A Desai, ... Nature Communications 12 (1), 6931, 2021 | 44 | 2021 |
LONP-1 and ATFS-1 sustain deleterious heteroplasmy by promoting mtDNA replication in dysfunctional mitochondria Q Yang, P Liu, NS Anderson, T Shpilka, YG Du, NU Naresh, R Li, LJ Zhu, ... Nature cell biology 24 (2), 181-193, 2022 | 39 | 2022 |
Orthogonal Cas9–Cas9 chimeras provide a versatile platform for genome editing MF Bolukbasi, P Liu, K Luk, SF Kwok, A Gupta, N Amrani, EJ Sontheimer, ... Nature communications 9 (1), 4856, 2018 | 39 | 2018 |
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 | 27 | 2021 |
Efficient homology-directed repair with circular single-stranded DNA donors S Iyer, A Mir, J Vega-Badillo, BP Roscoe, R Ibraheim, LJ Zhu, J Lee, P Liu, ... The CRISPR Journal 5 (5), 685-701, 2022 | 17 | 2022 |
Efficient homology-directed repair with circular ssDNA donors S Iyer, A Mir, J Vega-Badillo, BP Roscoe, R Ibraheim, LJ Zhu, J Lee, P Liu, ... bioRxiv, 864199, 2019 | 11 | 2019 |
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 | 7 | 2022 |
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 |
N1-arylation of 1, 4-benzodiazepine-2-ones with diaryliodonium salts R Khan, R Felix, PD Kemmitt, SJ Coles, GJ Tizzard, J Spencer Synlett 29 (02), 193-198, 2018 | 5 | 2018 |