| Adenovirus serotype 5 hexon mediates liver gene transfer SN Waddington, JH McVey, D Bhella, AL Parker, K Barker, H Atoda, ... Cell 132 (3), 397-409, 2008 | 728 | 2008 |
| Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and … AC Nathwani, JT Gray, CYC Ng, J Zhou, Y Spence, SN Waddington, ... Blood 107 (7), 2653-2661, 2006 | 519 | 2006 |
| Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins AC Nathwani, C Rosales, J McIntosh, G Rastegarlari, D Nathwani, D Raj, ... Molecular therapy 19 (5), 876-885, 2011 | 389 | 2011 |
| Mitochondria and quality control defects in a mouse model of Gaucher disease—links to Parkinson’s disease LD Osellame, AA Rahim, IP Hargreaves, ME Gegg, A Richard-Londt, ... Cell metabolism 17 (6), 941-953, 2013 | 371 | 2013 |
| Multiple vitamin K-dependent coagulation zymogens promote adenovirus-mediated gene delivery to hepatocytes AL Parker, SN Waddington, CG Nicol, DM Shayakhmetov, SM Buckley, ... Blood 108 (8), 2554-2561, 2006 | 335 | 2006 |
| Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant J McIntosh, PJ Lenting, C Rosales, D Lee, S Rabbanian, D Raj, N Patel, ... Blood, The Journal of the American Society of Hematology 121 (17), 3335-3344, 2013 | 315 | 2013 |
| Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice M Themis, SN Waddington, M Schmidt, C Von Kalle, Y Wang, F Al-Allaf, ... Molecular Therapy 12 (4), 763-771, 2005 | 282 | 2005 |
| Stable gene transfer to muscle using non-integrating lentiviral vectors L Apolonia, SN Waddington, C Fernandes, NJ Ward, G Bouma, ... Molecular Therapy 15 (11), 1947-1954, 2007 | 239 | 2007 |
| Codon optimization of human factor VIII cDNAs leads to high-level expression NJ Ward, SMK Buckley, SN Waddington, T VandenDriessche, ... Blood, The Journal of the American Society of Hematology 117 (3), 798-807, 2011 | 238 | 2011 |
| Identification of coagulation factor (F) X binding sites on the adenovirus serotype 5 hexon: effect of mutagenesis on FX interactions and gene transfer R Alba, AC Bradshaw, AL Parker, D Bhella, SN Waddington, SA Nicklin, ... Blood, The Journal of the American Society of Hematology 114 (5), 965-971, 2009 | 215 | 2009 |
| The CD46-Jagged1 interaction is critical for human TH1 immunity G Le Friec, D Sheppard, P Whiteman, CM Karsten, SAT Shamoun, ... Nature immunology 13 (12), 1213-1221, 2012 | 203 | 2012 |
| NRF2 orchestrates the metabolic shift during induced pluripotent stem cell reprogramming KE Hawkins, S Joy, JMKM Delhove, VN Kotiadis, E Fernandez, ... Cell reports 14 (8), 1883-1891, 2016 | 179 | 2016 |
| Fetal gene therapy for neurodegenerative disease of infants G Massaro, CNZ Mattar, AMS Wong, E Sirka, SMK Buckley, BR Herbert, ... Nature medicine 24 (9), 1317-1323, 2018 | 167 | 2018 |
| Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy SN Waddington, MS Nivsarkar, AR Mistry, SMK Buckley, G Kemball-Cook, ... Blood 104 (9), 2714-2721, 2004 | 152 | 2004 |
| Widespread Distribution and Muscle Differentiation of Human Fetal Mesenchymal Stem Cells After Intrauterine Transplantation in Dystrophic mdx Mouse J Chan, SN Waddington, K O'Donoghue, H Kurata, PV Guillot, ... Stem cells 25 (4), 875-884, 2007 | 150 | 2007 |
| Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects J Baruteau, SN Waddington, IE Alexander, P Gissen Journal of inherited metabolic disease 40 (4), 497-517, 2017 | 132 | 2017 |
| 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 |
| Activation and deactivation of periventricular white matter phagocytes during postnatal mouse development M Hristova, D Cuthill, V Zbarsky, A Acosta‐Saltos, A Wallace, K Blight, ... Glia 58 (1), 11-28, 2010 | 129 | 2010 |
| Genomic investigations of unexplained acute hepatitis in children S Morfopoulou, S Buddle, OE Torres Montaguth, L Atkinson, ... Nature 617 (7961), 564-573, 2023 | 126 | 2023 |
| In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor SN Waddington, SMK Buckley, M Nivsarkar, S Jezzard, H Schneider, ... Blood, The Journal of the American Society of Hematology 101 (4), 1359-1366, 2003 | 125 | 2003 |
Ahad RahimUniversity College LondonEmail được xác minh tại ucl.ac.uk