| Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells MD Hoban, GJ Cost, MC Mendel, Z Romero, ML Kaufman, AV Joglekar, ... Blood, The Journal of the American Society of Hematology 125 (17), 2597-2604, 2015 | 370 | 2015 |
| Site-specific gene editing of human hematopoietic stem cells for X-linked hyper-IgM syndrome CY Kuo, JD Long, B Campo-Fernandez, S de Oliveira, AR Cooper, ... Cell reports 23 (9), 2606-2616, 2018 | 136 | 2018 |
| β-globin gene transfer to human bone marrow for sickle cell disease Z Romero, F Urbinati, S Geiger, AR Cooper, J Wherley, ML Kaufman, ... The Journal of Clinical Investigation 123 (8), 3317-3330, 2013 | 113 | 2013 |
| Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency DA Carbonaro, L Zhang, X Jin, C Montiel-Equihua, S Geiger, M Carmo, ... Molecular Therapy 22 (3), 607-622, 2014 | 97 | 2014 |
| Improving gene editing outcomes in human hematopoietic stem and progenitor cells by temporal control of DNA repair A Lomova, DN Clark, B Campo-Fernandez, C Flores-Bjurström, ... Stem cells 37 (2), 284-294, 2019 | 90 | 2019 |
| Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells F Giannoni, CL Hardee, J Wherley, E Gschweng, S Senadheera, ... Molecular Therapy 21 (5), 1044-1054, 2013 | 71 | 2013 |
| Reactivating fetal hemoglobin expression in human adult erythroblasts through BCL11A knockdown using targeted endonucleases CF Bjurström, M Mojadidi, J Phillips, C Kuo, S Lai, GR Lill, A Cooper, ... Molecular therapy-nucleic acids 5, 2016 | 61 | 2016 |
| Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction DA Carbonaro, X Jin, X Wang, XJ Yu, N Rozengurt, ML Kaufman, X Wang, ... Blood, The Journal of the American Society of Hematology 120 (18), 3677-3687, 2012 | 48 | 2012 |
| Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells F Urbinati, PW Hargrove, S Geiger, Z Romero, J Wherley, ML Kaufman, ... Experimental hematology 43 (5), 346-351, 2015 | 43 | 2015 |
| HSV-sr39TK positron emission tomography and suicide gene elimination of human hematopoietic stem cells and their progeny in humanized mice EH Gschweng, MN McCracken, ML Kaufman, M Ho, RP Hollis, X Wang, ... Cancer research 74 (18), 5173-5183, 2014 | 38 | 2014 |
| Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study F Urbinati, B Campo Fernandez, KE Masiuk, V Poletti, RP Hollis, C Koziol, ... Human gene therapy 29 (10), 1153-1166, 2018 | 36 | 2018 |
| Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy K Baldwin, F Urbinati, Z Romero, B Campo-Fernandez, ML Kaufman, ... Stem Cells 33 (5), 1532-1542, 2015 | 34 | 2015 |
| Characterization of gene alterations following editing of the β-globin gene locus in hematopoietic stem/progenitor cells J Long, MD Hoban, AR Cooper, ML Kaufman, CY Kuo, ... Molecular therapy 26 (2), 468-479, 2018 | 25 | 2018 |
| Simultaneous deletion of Prdm1 and Vsx2 enhancers in the retina alters photoreceptor and bipolar cell fate specification, yet differs from deleting both genes NB Goodson, MA Kaufman, KU Park, JA Brzezinski IV Development 147 (13), dev190272, 2020 | 23 | 2020 |
| Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease F Urbinati, J Wherley, S Geiger, BC Fernandez, ML Kaufman, A Cooper, ... Cytotherapy 19 (9), 1096-1112, 2017 | 22 | 2017 |
| Transcriptional profiling of murine retinas undergoing semi-synchronous cone photoreceptor differentiation ML Kaufman, KU Park, NB Goodson, S Chew, S Bersie, KL Jones, ... Developmental biology 453 (2), 155-167, 2019 | 20 | 2019 |
| The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells Z Romero, B Campo-Fernandez, J Wherley, ML Kaufman, F Urbinati, ... Molecular Therapy Methods & Clinical Development 2, 2015 | 20 | 2015 |
| Initiation of Otx2 expression in the developing mouse retina requires a unique enhancer and either Ascl1 or Neurog2 activity ML Kaufman, NB Goodson, KU Park, M Schwanke, E Office, ... Development 148 (12), dev199399, 2021 | 16 | 2021 |
| Dosing and re-administration of lentiviral vector for in vivo gene therapy in rhesus monkeys and ADA-deficient mice DA Carbonaro-Sarracino, AF Tarantal, CCI Lee, ML Kaufman, S Wandro, ... Molecular Therapy-Methods & Clinical Development 16, 78-93, 2020 | 11 | 2020 |
| Office E, Schneider SR, Abraham J, Hensley A, Jones KL, Brzezinski JA (2021) Initiation of Otx2 expression in the developing mouse retina requires a unique enhancer and either … ML Kaufman, NB Goodson, KU Park, M Schwanke Development 148, 0 | 6 | |
