UCL Great Ormond Street Institute of Child Health


Great Ormond Street Institute of Child Health


Dr Simon Waddington

Dr Simon Waddington

Nivsarkar MS, Buckley SM, Parker AL, Perocheau D, McKay TR, Rahim AA, Howe SJ, Waddington SN. Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector delivery. J Immunol Res 2015; 2015: 397879

Mattar CN, Wong AM, Hoefer K, Alonso-Ferrero ME, Buckley SM, Howe SJ, Cooper JD, Waddington SN, Chan JK, Rahim AA. Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates. FASEB J 2015

Buckley SM, Delhove JM, Perocheau DP, Karda R, Rahim AA, Howe SJ, Ward NJ, Birrell MA, Belvisi MG, Arbuthnot P, Johnson MR, Waddington SN, McKay TR. In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters. Sci Rep 2015; 5: 11842

Buckinx R, Van Remoortel S, Gijsbers R, Waddington SN, Timmermans JP. Proof-of-concept: neonatal intravenous injection of adeno-associated virus vectors results in successful transduction of myenteric and submucosal neurons n the mouse small and large intestine. Neurogastroent Motil 2015; In Press.

Karda R, Buckley SM, Mattar CN, Ng J, Massaro G, Hughes MP, Kurian MA, Baruteau J, Gissen P, Chan JK, Bacchelli C, Waddington SN, Rahim AA. Perinatal systemic gene delivery using adeno-associated viral vectors. Front Mol Neurosci 2014; 7: 89

Jonuschies J, Antoniou M, Waddington S, Boldrin L, Muntoni F, Thrasher A, Morgan J. The human desmin promoter drives robust gene expression for skeletal muscle stem cell-mediated gene therapy. Curr Gene Ther 2014; 14: 276-288

Di Matteo M, Samara-Kuko E, Ward NJ, Waddington SN, McVey JH, Chuah MK, VandenDriessche T. Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy. Mol Ther 2014; 22: 1614-1624

Osellame LD, Rahim AA, Hargreaves IP, Gegg ME, Richard-Londt A, Brandner S, Waddington SN, Schapira AH, Duchen MR. Mitochondria and quality control defects in a mouse model of Gaucher disease--links to Parkinson's disease. Cell Metab 2013; 17: 941-953.

McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood 2013; 121: 3335-3344.

Mattar CN, Waddington SN, Biswas A, Johana N, Ng XW, Fisk AS, Fisk NM, Tan LG, Rahim AA, Buckley SM, Tan MH, Lu J, Choolani M, Chan JK. Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems. Gene Ther 2013; 20: 69-83.

Rahim AA, Wong AM, Ahmadi S, Hoefer K, Buckley SM, Hughes DA, Nathwani AN, Baker AH, McVey JH, Cooper JD, Waddington SN. In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression. Gene Ther 2012; 19: 936-946.

Le Friec G, Sheppard D, Whiteman P, Karsten CM, Shamoun SA, Laing A, Bugeon L, Dallman MJ, Melchionna T, Chillakuri C, Smith RA, Drouet C, Couzi L, Fremeaux-Bacchi V, Kohl J, Waddington SN, McDonnell JM, Baker A, Handford PA, Lea SM, Kemper C. The CD46-Jagged1 interaction is critical for human T(H)1 immunity. Nat Immunol 2012; 13: 1213-1221.

inny CJ, McIntosh J, Della Peruta M, Kymalainen H, Tuddenham EGD, Buckley SMK, Waddington SN, McVey JH, Spence Y, Morton CL, Thrasher AJ, Gray JT, Castellino FJ, Tarantal AF, Davidoff AM, Nathwani AC. AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage. Blood 2012; 119: 957-966.

Ward NJ, Buckley SMK, Waddington SN, VandenDriessche T, Chuah MK, Nathwani AC, McIntosh J, Tuddenham EGD, Kinnon C, Thrasher AJ, McVey JH. Codon optimisation of human factor VIII cDNAs leads to high level expression. Blood 2011; 117: 798-807.

Rahim AA, Wong AMS, Hoefer K, Buckley SMK, Mattar CN, Cheng SH, Chan JKY, Cooper JD, Waddington SN. Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system. FASEB J 2011; 25: 3505-3518.

Rahim AA, Buckley SMK, Chan JKY, Peebles DM, Waddington SN. Perinatal gene delivery to the CNS. Ther Deliv 2011; 2: 483-491.

Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM. 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. Mol Ther 2011; 19: 876-885.

Mattar CN, Nathwani AC, Waddington SN, Dighe N, Kaeppel C, Nowrouzi A, McIntosh J, Johana NB, Ogden B, Fisk NM, Davidoff AM, David A, Peebles D, Valentine MB, Appelt JU, von Kalle C, Schmidt M, Biswas A, Choolani M, Chan JK. Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques. Mol Ther 2011; 19: 1950-1960.

Grinshpun A, Condiotti R, Waddington S, Peer M, Zeig E, Peretz S, Simerzin A, Chou J, Pann C-J, Giladi H, Galun E. Neonatal gene therapy of glycogen storage disease type Ia using a feline immunodeficiency virus based vector. Mol Ther 2010; 18: 1592-1598.

Waddington SN, McVey JH, Bhella D, Parker AL, Barker K, Atoda H, Pink R, Buckley SMK, Greig JA, Denby L, Custers J, Morita T, Francischetti IMB, Monteiro RQ, Barouch DH, van Rooijen N, Napoli C, Havenga MJE, Nicklin SA, Baker AH. Adenovirus serotype 5 hexon mediates liver gene transfer. Cell 2008; 132: 397-409