Simon N Waddington
Simon N Waddington
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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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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