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 | 382 | 2015 |
CRISPR/Cas9-mediated correction of the sickle mutation in human CD34+ cells MD Hoban, D Lumaquin, CY Kuo, Z Romero, J Long, M Ho, CS Young, ... Molecular Therapy 24 (9), 1561-1569, 2016 | 230 | 2016 |
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 | 142 | 2018 |
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 | 62 | 2016 |
Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients AR Cooper, GR Lill, K Shaw, DA Carbonaro-Sarracino, A Davila, ... Blood, The Journal of the American Society of Hematology 129 (19), 2624-2635, 2017 | 36 | 2017 |
Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter AR Cooper, GR Lill, EH Gschweng, DB Kohn Nucleic acids research 43 (1), 682-690, 2015 | 20 | 2015 |
Genetic tagging during human mesoderm differentiation reveals tripotent lateral plate mesodermal progenitors CJ Chin, AR Cooper, GR Lill, D Evseenko, Y Zhu, CB He, D Casero, ... Stem cells 34 (5), 1239-1250, 2016 | 16 | 2016 |
RNA isolation from fetal and adult human tissues for transcriptional profiling M Votteler, SL Layland, G Lill, KGM Brockbank, A Horke, ... Biotechnology Journal 8 (3), 338-344, 2013 | 5 | 2013 |
Targeted Gene Insertion for the Treatment of X-Linked Hyper-IgM Syndrome CY Kuo, JD Long, B Campo-Fernandez, S de Oliveira, AR Cooper, ... MOLECULAR THERAPY 26 (5), 373-373, 2018 | | 2018 |
Barcoding as a Tool To Predict Integrating Vector Genotoxicity in HSPCs AR Cooper, KL Shaw, GR Lill, R Sokolic, F Candotti, DB Kohn MOLECULAR THERAPY 23, S94-S95, 2015 | | 2015 |
115. Correction of the Sickle-Cell Disease Mutation in Human Hematopoietic Stem/Progenitor Cells MD Hoban, MC Mendel, Z Romero, ML Kaufman, AV Joglekar, M Ho, ... Molecular Therapy 23, S48, 2015 | | 2015 |
240. Stable and Clinically Benign Clonal Dominance in an ADA-SCID Patient Treated With Retroviral Gene Therapy AR Cooper, KL Shaw, GR Lill, R Sokolic, F Candotti, DB Kohn Molecular Therapy 23, S94, 2015 | | 2015 |