| Gene therapy comes of age CE Dunbar, KA High, JK Joung, DB Kohn, K Ozawa, M Sadelain Science 359 (6372), eaan4672, 2018 | 1244 | 2018 |
| Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo N Holt, J Wang, K Kim, G Friedman, X Wang, V Taupin, GM Crooks, ... Nature biotechnology 28 (8), 839-847, 2010 | 828 | 2010 |
| Transplantation outcomes for severe combined immunodeficiency, 2000–2009 SY Pai, BR Logan, LM Griffith, RH Buckley, RE Parrott, CC Dvorak, ... New England Journal of Medicine 371 (5), 434-446, 2014 | 741 | 2014 |
| Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States A Kwan, RS Abraham, R Currier, A Brower, K Andruszewski, JK Abbott, ... Jama 312 (7), 729-738, 2014 | 721 | 2014 |
| Engraftment of gene–modified umbilical cord blood cells in neonates with adenosine deaminase deficiency DB Kohn, KI Weinberg, JA Nolta, LN Heiss, C Lenarsky, GM Crooks, ... Nature medicine 1 (10), 1017-1023, 1995 | 701 | 1995 |
| Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. PM Challita, DB Kohn Proceedings of the National Academy of Sciences 91 (7), 2567-2571, 1994 | 533 | 1994 |
| Stable transduction of quiescent CD34+CD38− human hematopoietic cells by HIV-1-based lentiviral vectors SS Case, MA Price, CT Jordan, XJ Yu, L Wang, G Bauer, DL Haas, D Xu, ... Proceedings of the National Academy of Sciences 96 (6), 2988-2993, 1999 | 530 | 1999 |
| Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells MA DeWitt, W Magis, NL Bray, T Wang, JR Berman, F Urbinati, SJ Heo, ... Science translational medicine 8 (360), 360ra134-360ra134, 2016 | 504 | 2016 |
| Occurrence of leukaemia following gene therapy of X-linked SCID DB Kohn, M Sadelain, JC Glorioso Nature Reviews Cancer 3 (7), 477-488, 2003 | 460 | 2003 |
| A modified γ-retrovirus vector for X-linked severe combined immunodeficiency S Hacein-Bey-Abina, SY Pai, HB Gaspar, M Armant, CC Berry, S Blanche, ... New England Journal of Medicine 371 (15), 1407-1417, 2014 | 458 | 2014 |
| Analyzing CRISPR genome-editing experiments with CRISPResso L Pinello, MC Canver, MD Hoban, SH Orkin, DB Kohn, DE Bauer, ... Nature biotechnology 34 (7), 695-697, 2016 | 444 | 2016 |
| Immune response to green fluorescent protein: implications for gene therapy R Stripecke, M del Carmen Villacres, DC Skelton, N Satake, S Halene, ... Gene therapy 6 (7), 1305-1312, 1999 | 414 | 1999 |
| A single CRISPR-Cas9 deletion strategy that targets the majority of DMD patients restores dystrophin function in hiPSC-derived muscle cells CS Young, MR Hicks, NV Ermolova, H Nakano, M Jan, S Younesi, ... Cell stem cell 18 (4), 533-540, 2016 | 404 | 2016 |
| T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates DB Kohn, MS Hershfield, D Carbonaro, A Shigeoka, J Brooks, ... Nature medicine 4 (7), 775-780, 1998 | 388 | 1998 |
| 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 | 369 | 2015 |
| Genetic therapies against HIV JJ Rossi, CH June, DB Kohn Nature biotechnology 25 (12), 1444-1454, 2007 | 332 | 2007 |
| Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience WT Shearer, E Dunn, LD Notarangelo, CC Dvorak, JM Puck, BR Logan, ... Journal of allergy and clinical immunology 133 (4), 1092-1098, 2014 | 323 | 2014 |
| Sustained human hematopoiesis in immunodeficient mice by cotransplantation of marrow stroma expressing human interleukin-3: analysis of gene transduction of long-lived progenitors JA Nolta, MB Hanley, DB Kohn | 316 | 1994 |
| Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system H Singh, PR Manuri, S Olivares, N Dara, MJ Dawson, H Huls, PB Hackett, ... Cancer research 68 (8), 2961-2971, 2008 | 314 | 2008 |
| A Clinical Trial of Retroviral-Mediated Transfer of arev-Responsive Element Decoy Gene Into CD34+Cells From the Bone Marrow of Human Immunodeficiency … DB Kohn, G Bauer, CR Rice, JC Rothschild, DA Carbonaro, P Valdez, ... Blood, The Journal of the American Society of Hematology 94 (1), 368-371, 1999 | 295 | 1999 |
Jan NoltaProfessor, Director of the Stem Cell Program, University of California DavisVerified email at ucdavis.edu
