| 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 |
| High-efficiency lentiviral transduction of human CD34+ cells in high-density culture with poloxamer and prostaglandin E2 N Uchida, T Nassehi, CM Drysdale, J Gamer, M Yapundich, S Demirci, ... Molecular Therapy-Methods & Clinical Development 13, 187-196, 2019 | 41 | 2019 |
| Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease N Uchida, CM Drysdale, T Nassehi, J Gamer, M Yapundich, J DiNicola, ... Molecular Therapy-Methods & Clinical Development 21, 121-132, 2021 | 31 | 2021 |
| Busulfan combined with immunosuppression allows efficient engraftment of gene-modified cells in a rhesus macaque model N Uchida, T Nassehi, CM Drysdale, J Gamer, M Yapundich, ... Molecular Therapy 27 (9), 1586-1596, 2019 | 29 | 2019 |
| Hematopoietic stem cell-targeted gene-addition and gene-editing strategies for β-hemoglobinopathies CM Drysdale, T Nassehi, J Gamer, M Yapundich, JF Tisdale, N Uchida Cell Stem Cell 28 (2), 191-208, 2021 | 24 | 2021 |
| Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models N Uchida, L Li, T Nassehi, CM Drysdale, M Yapundich, J Gamer, ... Cell Reports Medicine 2 (4), 2021 | 23 | 2021 |
| Low-dose busulfan reduces human CD34+ cell doses required for engraftment in c-kit mutant immunodeficient mice A Leonard, M Yapundich, T Nassehi, J Gamer, CM Drysdale, ... Molecular Therapy-Methods & Clinical Development 15, 430-437, 2019 | 23 | 2019 |
| βT87Q-globin gene therapy reduces sickle hemoglobin production, allowing for ex vivo anti-sickling activity in human erythroid cells S Demirci, B Gudmundsdottir, Q Li, JJ Haro-Mora, T Nassehi, C Drysdale, ... Molecular Therapy Methods & Clinical Development 17, 912-921, 2020 | 15 | 2020 |
| Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor N Uchida, F Ferrara, CM Drysdale, M Yapundich, J Gamer, T Nassehi, ... Science Translational Medicine 13 (591), eabb0411, 2021 | 8 | 2021 |
| 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 |
| Fetal hemoglobin and F-cell variance in mobilized CD34+ cell-transplanted rhesus monkeys S Demirci, JJH Mora, M Yapundich, C Drysdale, J Gamer, T Nassehi, ... Experimental hematology 75, 21-25. e1, 2019 | 4 | 2019 |
| Ex vivo immunological evaluation of stable mixed chimeric patients after matched related donor allogeneic transplantation in sickle cell disease LN Raines, MM Hsieh, T Nassehi, CM Drysdale, JF Tisdale, N Uchida Cytotherapy 21 (12), 1206-1215, 2019 | 3 | 2019 |
| Preclinical evaluation for engraftment of gene-edited CD34+ cells with a sickle cell disease mutation in a rhesus transplantation model N Uchida, L Li, T Nassehi, M Yapundich, J Gamer, C Drysdale, ... Blood 134, 609, 2019 | 3 | 2019 |
| Development of Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease N Uchida, T Nassehi, CM Drysdale, J Gamer, M Yapundich, S Demirci, ... Molecular Therapy 28 (4), 395-396, 2020 | 2 | 2020 |
| Truncated erythropoietin receptors confer an in vivo selective advantage in gene-modified erythroid cells expressing fetal hemoglobin due to bcl11a interference N Uchida, C Drysdale, M Yapundich, J Gamer, T Nassehi, M Wielgosz, ... Blood 134, 2063, 2019 | 2 | 2019 |
| Development of a clinically applicable method for high-efficiency gene correction of plerixafor-mobilized CD34+ cells from patients with sickle cell disease N Uchida, L Li, JJ Haro-Mora, S Demirci, T Nassehi, J Gamer, C Drysdale, ... Blood 132, 2191, 2018 | 2 | 2018 |
| Durable robust fetal globin induction in rhesus monkeys following BCL11A erythroid enhancer edited autologous hematopoietic stem cell transplant S Demirci, J Zeng, Y Wu, N Uchida, J Gamer, M Yapundich, C Drysdale, ... MOLECULAR THERAPY 28 (4), 241-242, 2020 | 1 | 2020 |
| Engraftability of gene-edited sickle cell disease CD34+ cells in xenograft mouse and rhesus transplantation models N Uchida, L Li, T Nassehi, M Yapundich, J Gamer, C Drysdale, ... MOLECULAR THERAPY 28 (4), 175-175, 2020 | 1 | 2020 |
| Let us know how access to this document benefits you. S Demirci, J Zeng, Y Wu, N Uchida, AH Shen, D Pellin, J Gamer, ... | | 2020 |
| High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Cell Culture with Poloxamer and Prostaglandin E2 Supplementation N Uchida, T Nassehi, CM Drysdale, J Gamer, M Yapundich, S Demirci, ... MOLECULAR THERAPY 27 (4), 325-325, 2019 | | 2019 |
