| Effect of gene therapy on visual function in Leber's congenital amaurosis JWB Bainbridge, AJ Smith, SS Barker, S Robbie, R Henderson, ... New England Journal of Medicine 358 (21), 2231-2239, 2008 | 2220 | 2008 |
| Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy S Hacein-Bey-Abina, F Le Deist, F Carlier, C Bouneaud, C Hue, ... New england journal of medicine 346 (16), 1185-1193, 2002 | 1367 | 2002 |
| Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 MG Ott, M Schmidt, K Schwarzwaelder, S Stein, U Siler, U Koehl, H Glimm, ... Nature medicine 12 (4), 401-409, 2006 | 1296 | 2006 |
| 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 | 1258 | 2008 |
| Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease S Stein, MG Ott, S Schultze-Strasser, A Jauch, B Burwinkel, A Kinner, ... Nature medicine 16 (2), 198-204, 2010 | 814 | 2010 |
| 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 | 773 | 2004 |
| Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells W Qasim, H Zhan, S Samarasinghe, S Adams, P Amrolia, S Stafford, ... Science translational medicine 9 (374), eaaj2013, 2017 | 727 | 2017 |
| High-level transduction and gene expression in hematopoietic repopulating cells using a human imunodeficiency virus type 1-based lentiviral vector containing an internal spleen … C Demaison, K Parsley, G Brouns, M Scherr, K Battmer, C Kinnon, M Grez, ... Human gene therapy 13 (7), 803-813, 2002 | 583 | 2002 |
| The cytoplasm of living cells behaves as a poroelastic material E Moeendarbary, L Valon, M Fritzsche, AR Harris, DA Moulding, ... Nature materials 12 (3), 253-261, 2013 | 554 | 2013 |
| 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 | 523 | 2006 |
| A robust model for read count data in exome sequencing experiments and implications for copy number variant calling V Plagnol, J Curtis, M Epstein, KY Mok, E Stebbings, S Grigoriadou, ... Bioinformatics 28 (21), 2747-2754, 2012 | 514 | 2012 |
| The wiskott-aldrich syndrome HD Ochs, AJ Thrasher Journal of Allergy and Clinical Immunology 117 (4), 725-738, 2006 | 473 | 2006 |
| Clinical features and outcome of patients with IRAK-4 and MyD88 deficiency C Picard, H Von Bernuth, P Ghandil, M Chrabieh, O Levy, PD Arkwright, ... Medicine 89 (6), 403, 2010 | 420 | 2010 |
| Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors U Modlich, S Navarro, D Zychlinski, T Maetzig, S Knoess, MH Brugman, ... Molecular Therapy 17 (11), 1919-1928, 2009 | 411 | 2009 |
| WASP: a key immunological multitasker AJ Thrasher, SO Burns Nature Reviews Immunology 10 (3), 182-192, 2010 | 402 | 2010 |
| 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 | 383 | 2014 |
| Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy RR Ali, GM Sarra, C Stephens, M Alwis, JWB Bainbridge, PM Munro, ... Nature genetics 25 (3), 306-310, 2000 | 374 | 2000 |
| Configuration of human dendritic cell cytoskeleton by Rho GTPases, the WAS protein, and differentiation S Burns, AJ Thrasher, MP Blundell, L Machesky, GE Jones Blood, The Journal of the American Society of Hematology 98 (4), 1142-1149, 2001 | 369 | 2001 |
| Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy F Eichler, C Duncan, PL Musolino, PJ Orchard, S De Oliveira, AJ Thrasher, ... New England Journal of Medicine 377 (17), 1630-1638, 2017 | 367 | 2017 |
| Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome SHB Abina, HB Gaspar, J Blondeau, L Caccavelli, S Charrier, K Buckland, ... Jama 313 (15), 1550-1563, 2015 | 357 | 2015 |
