| Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients SJ Howe, MR Mansour, K Schwarzwaelder, C Bartholomae, M Hubank, ... The Journal of clinical investigation 118 (9), 2008 | 1454 | 2008 |
| Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector HB Gaspar, KL Parsley, S Howe, D King, KC Gilmour, J Sinclair, G Brouns, ... The Lancet 364 (9452), 2181-2187, 2004 | 837 | 2004 |
| Effective gene therapy with nonintegrating lentiviral vectors RJ Yánez-Munoz, KS Balaggan, A MacNeil, SJ Howe, M Schmidt, ... Nature medicine 12 (3), 348-353, 2006 | 570 | 2006 |
| Hot spots of retroviral integration in human CD34+ hematopoietic cells C Cattoglio, G Facchini, D Sartori, A Antonelli, A Miccio, B Cassani, ... Blood, The Journal of the American Society of Hematology 110 (6), 1770-1778, 2007 | 336 | 2007 |
| Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy A Deichmann, S Hacein-Bey-Abina, M Schmidt, A Garrigue, MH Brugman, ... The Journal of clinical investigation 117 (8), 2225-2232, 2007 | 298 | 2007 |
| Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency HB Gaspar, S Cooray, KC Gilmour, KL Parsley, S Adams, SJ Howe, ... Science translational medicine 3 (97), 97ra79-97ra79, 2011 | 280 | 2011 |
| Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo K Schwarzwaelder, SJ Howe, M Schmidt, MH Brugman, A Deichmann, ... The Journal of clinical investigation 117 (8), 2241-2249, 2007 | 242 | 2007 |
| Comprehensive genomic access to vector integration in clinical gene therapy R Gabriel, R Eckenberg, A Paruzynski, CC Bartholomae, A Nowrouzi, ... Nature medicine 15 (12), 1431-1436, 2009 | 221 | 2009 |
| Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic … F Zhang, SI Thornhill, SJ Howe, M Ulaganathan, A Schambach, J Sinclair, ... Blood, The Journal of the American Society of Hematology 110 (5), 1448-1457, 2007 | 219 | 2007 |
| Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency SI Thornhill, A Schambach, SJ Howe, M Ulaganathan, E Grassman, ... Molecular therapy 16 (3), 590-598, 2008 | 193 | 2008 |
| Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy M Tschernutter, FC Schlichtenbrede, S Howe, KS Balaggan, PM Munro, ... Gene therapy 12 (8), 694-701, 2005 | 174 | 2005 |
| Failure of SCID-X1 gene therapy in older patients AJ Thrasher, S Hacein-Bey-Abina, HB Gaspar, S Blanche, EG Davies, ... Blood 105 (11), 4255-4257, 2005 | 145 | 2005 |
| Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector O Argyros, SP Wong, M Niceta, SN Waddington, SJ Howe, C Coutelle, ... Gene therapy 15 (24), 1593-1605, 2008 | 130 | 2008 |
| Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice A Langford-Smith, FL Wilkinson, KJ Langford-Smith, RJ Holley, ... Molecular Therapy 20 (8), 1610-1621, 2012 | 121 | 2012 |
| Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors AA Rahim, AMS Wong, SJ Howe, SMK Buckley, AD Acosta-Saltos, ... Gene therapy 16 (4), 509-520, 2009 | 114 | 2009 |
| Retrovirus and lentivirus vector design and methods of cell conditioning S Cooray, SJ Howe, AJ Thrasher Methods in enzymology 507, 29-57, 2012 | 106 | 2012 |
| Ectopic retroviral expression of LMO2, but not IL2Rγ, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy K Pike-Overzet, D de Ridder, F Weerkamp, MRM Baert, MMA Verstegen, ... Leukemia 21 (4), 754-763, 2007 | 89 | 2007 |
| Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver O Argyros, SP Wong, C Fedonidis, O Tolmachov, SN Waddington, ... Journal of molecular medicine 89, 515-529, 2011 | 85 | 2011 |
| Minicircle DNA provides enhanced and prolonged transgene expression following airway gene transfer MM Munye, AD Tagalakis, JL Barnes, RE Brown, RJ McAnulty, SJ Howe, ... Scientific reports 6 (1), 23125, 2016 | 83 | 2016 |
| Lentiviral vectors can be used for full-length dystrophin gene therapy JR Counsell, Z Asgarian, J Meng, V Ferrer, CA Vink, SJ Howe, ... Scientific reports 7 (1), 44775, 2017 | 82 | 2017 |
Simon N WaddingtonUniversity College London在 ucl.ac.uk 的电子邮件经过验证
