Dr Simon Waddington, BSc MSc PhD - University College London - Reader in Gene Transfer Technology

Dr Simon Waddington

BSc MSc PhD

University College London

Reader in Gene Transfer Technology

London, Greater London | United Kingdom

Main Specialties: Medical Genetics

Additional Specialties: Gene Therapy


Top Author

Dr Simon Waddington, BSc MSc PhD - University College London - Reader in Gene Transfer Technology

Dr Simon Waddington

BSc MSc PhD

Introduction

I am Joint Acting Director of the Institute for Women’s Health and also lead a team which is focused upon preclinical models of gene and stem cell transfer, particularly for perinatal gene delivery for early onset, debilitating and often lethal diseases including neurodegenerative diseases, inherited epilepsy and metabolic diseases. For Dravet Syndrome we recently obtained funding from the UK Medical Research Council Developmental Pathway Funding Scheme. We have obtained project grant funding for neuronopathic Gaucher Disease and, led by Dr Ahad Rahim, Niemann Pick Type C at UCL School of Pharmacy.
In a parallel research stream, we have developed an application of gene transfer, with Prof Tristan McKay, whereby signalling pathways in diseased organs and tumours can be quantified continually and non-invasively. This was funded, primarily, through a European Research Council Starting Grant. From this work we filed two patents through University College London.
In collaboration with Professors Andy Baker and John McVey we elucidated one of the fundamental mechanisms by which adenovirus vectors transduce cells and published this work in Cell journal. My team and I have published more than one hundred peer reviewed publications and reviews in journals including Nature Immunology, Cell Metabolism and Blood.
The expertise of my team and I in mouse models of perinatal intervention has enabled us to maintain excellent collaborations in areas such as preterm labour and perinatal brain injury, with Professors Donald Peebles, Mark Johnson and Phil Bennet. We have several long-standing international collaborations. These include Drs Jerry Chan and Citra Mattar, Duke-NUS Graduate Medical School Singapore and Prof Patrick Arbuthnot, at the University of Witwatersrand, South Africa, where I hold an honorary position.

Primary Affiliation: University College London - London, Greater London , United Kingdom

Specialties:

Additional Specialties:

Education

Jan 1999
Imperial College London
PhD

Publications

82Publications

1572Reads

756Profile Views

1755PubMed Central Citations

Vps33b is crucial for structural and functional hepatocyte polarity.

Authors:
Joanna Hanley Dipok Kumar Dhar Francesca Mazzacuva Rebeca Fiadeiro Jemima J Burden Anne-Marie Lyne Holly Smith Anna Straatman-Iwanowska Blerida Banushi Alex Virasami Kevin Mills Frédéric P Lemaigre A S Knisely Steven Howe Neil Sebire Simon N Waddington Coen C Paulusma Peter Clayton Paul Gissen

J Hepatol 2017 05 9;66(5):1001-1011. Epub 2017 Jan 9.

UCL Institute of Child Health, University College London, London WC1N 1EH, UK; MRC Laboratory for Molecular Cell Biology, University College London, London WC1E 6BT, UK; Inherited Metabolic Disease Unit, Great Ormond Street Hospital for Children NHS Trust, London WC1N 3JH, UK. Electronic address:

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http://dx.doi.org/10.1016/j.jhep.2017.01.001DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5387182PMC
May 2017
15 Reads
3 Citations
11.340 Impact Factor

The Local and Systemic Immune Response to Intrauterine LPS in the Prepartum Mouse.

Authors:
Lydia F Edey Kieran P O'Dea Bronwen R Herbert Renyi Hua Simon N Waddington David A MacIntyre Philip R Bennett Masao Takata Mark R Johnson

Biol Reprod 2016 12 19;95(6):125. Epub 2016 Oct 19.

Imperial College Parturition Research Group, Academic Department of Obstetrics & Gynaecology, Imperial College School of Medicine, Chelsea and Westminster Hospital, London, United Kingdom

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http://dx.doi.org/10.1095/biolreprod.116.143289DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5333944PMC
December 2016
8 Reads
1 Citation
3.320 Impact Factor

Haemophilia B curative FIX production from a low dose UCOE-based lentiviral vector following hepatic pre-natal delivery.

Authors:
Vincent Yu-Cheng Kao Sonia Ferreira Simon Nicholas Waddington Michael Nicholas Antoniou

Curr Gene Ther 2016 Nov 2. Epub 2016 Nov 2.

Gene Expression and Therapy Group, King`s College London, Faculty of Life Sciences and Medicine, Department of Medical and Molecular Genetics, 8th Floor Tower Wing, Guy's Hospital, London SE1 9RT, UK.

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November 2016
25 Reads
2.542 Impact Factor

Regulation of post-Golgi LH3 trafficking is essential for collagen homeostasis.

Authors:
Blerida Banushi Federico Forneris Anna Straatman-Iwanowska Adam Strange Anne-Marie Lyne Clare Rogerson Jemima J Burden Wendy E Heywood Joanna Hanley Ivan Doykov Kornelis R Straatman Holly Smith Danai Bem Janos Kriston-Vizi Gema Ariceta Maija Risteli Chunguang Wang Rosalyn E Ardill Marcin Zaniew Julita Latka-Grot Simon N Waddington S J Howe Francesco Ferraro Asllan Gjinovci Scott Lawrence Mark Marsh Mark Girolami Laurent Bozec Kevin Mills Paul Gissen

Nat Commun 2016 07 20;7:12111. Epub 2016 Jul 20.

MRC Laboratory for Molecular Cell Biology, University College London, London WC1E 6BT, UK.

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http://dx.doi.org/10.1038/ncomms12111DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4961739PMC
July 2016
25 Reads
8 Citations
10.742 Impact Factor

Modeling hormonal and inflammatory contributions to preterm and term labor using uterine temporal transcriptomics.

Authors:
Roberta Migale David A MacIntyre Stefano Cacciatore Yun S Lee Henrik Hagberg Bronwen R Herbert Mark R Johnson Donald Peebles Simon N Waddington Phillip R Bennett

BMC Med 2016 06 13;14(1):86. Epub 2016 Jun 13.

Imperial College Parturition Research Group, Institute of Reproduction and Developmental Biology, Imperial College London, Hammersmith Campus, London, United Kingdom.

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http://dx.doi.org/10.1186/s12916-016-0632-4DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4904357PMC
June 2016
25 Reads
3 Citations
7.250 Impact Factor

NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming.

Authors:
Kate E Hawkins Shona Joy Juliette M K M Delhove Vassilios N Kotiadis Emilio Fernandez Lorna M Fitzpatrick James R Whiteford Peter J King Juan P Bolanos Michael R Duchen Simon N Waddington Tristan R McKay

Cell Rep 2016 Mar 18;14(8):1883-91. Epub 2016 Feb 18.

Stem Cell Group, Cardiovascular and Cell Sciences Research Institute, St. George's University of London, Cranmer Terrace, London SW17 0RE, UK; School of Healthcare Science, John Dalton Building, Manchester Metropolitan University, Chester Street, Manchester M1 5GD, UK. Electronic address:

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http://dx.doi.org/10.1016/j.celrep.2016.02.003DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4785773PMC
March 2016
52 Reads
21 Citations
7.210 Impact Factor

Gene Therapy with Adeno-associated Virus for Cystic Fibrosis.

Authors:
Rajvinder Karda Suzanne M K Buckley Simon N Waddington

Am J Respir Crit Care Med 2016 Feb;193(3):234-6

2 Gene Transfer Technology Group University College London London, United Kingdom.

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http://dx.doi.org/10.1164/rccm.201510-2024EDDOI Listing
February 2016
18 Reads
1 Citation
13.000 Impact Factor

A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells.

Authors:
Simon N Waddington Riccardo Privolizzi Rajvinder Karda Helen C O'Neill

Curr Stem Cell Rep 2016;2:9-20. Epub 2016 Feb 11.

Preimplantation Genetics and Embryology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX UK.

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http://dx.doi.org/10.1007/s40778-016-0037-5DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4913977PMC
February 2016
10 Reads
7 Citations

Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector delivery.

Authors:
Megha S Nivsarkar Suzanne M K Buckley Alan L Parker Dany Perocheau Tristan R McKay Ahad A Rahim Steven J Howe Simon N Waddington

J Immunol Res 2015 31;2015:397879. Epub 2015 Jan 31.

Gene Transfer Technology Group, University College London, 86-96 Chenies Mews, London WC1E 6HXZ, UK ; Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.

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http://dx.doi.org/10.1155/2015/397879DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4352475PMC
December 2015
35 Reads
4 Citations

Specific inhibition of c-Jun N-terminal kinase delays preterm labour and reduces mortality.

Authors:
Grisha Pirianov David A MacIntyre Yun Lee Simon N Waddington Vasso Terzidou Huseyin Mehmet Phillip R Bennett

Reproduction 2015 Oct 16;150(4):269-77. Epub 2015 Jul 16.

Imperial College Parturition Research GroupDepartment of Reproductive Biology, Institute of Reproductive and Developmental Biology, Imperial College London, Hammersmith Campus, Du Cane Road, London W12 0NN, UKGene Transfer Technology GroupInstitute for Women's Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, UKProteostasis Therapeutics 200 Technology SquareSuite 402, Cambridge, Massachusetts 02139, USADepartment of Biomedical and Forensic SciencesAnglia Ruskin University, East Road, Cambridge CB1 1PT, UK.

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http://dx.doi.org/10.1530/REP-15-0258DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4982111PMC
October 2015
15 Reads
2 Citations
3.174 Impact Factor

Specific Lipopolysaccharide Serotypes Induce Differential Maternal and Neonatal Inflammatory Responses in a Murine Model of Preterm Labor.

Authors:
Roberta Migale Bronwen R Herbert Yun S Lee Lynne Sykes Simon N Waddington Donald Peebles Henrik Hagberg Mark R Johnson Phillip R Bennett David A MacIntyre

Am J Pathol 2015 Sep 26;185(9):2390-401. Epub 2015 Jul 26.

Imperial College Parturition Research Group, Institute of Reproductive and Developmental Biology, Imperial College London, London, United Kingdom. Electronic address:

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https://linkinghub.elsevier.com/retrieve/pii/S00029440150036
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http://dx.doi.org/10.1016/j.ajpath.2015.05.015DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4597270PMC
September 2015
18 Reads
7 Citations
4.591 Impact Factor

Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates.

Authors:
Citra N Mattar Andrew M S Wong Klemens Hoefer Maria E Alonso-Ferrero Suzanne M K Buckley Steven J Howe Jonathan D Cooper Simon N Waddington Jerry K Y Chan Ahad A Rahim

FASEB J 2015 Sep 10;29(9):3876-88. Epub 2015 Jun 10.

*Experimental Fetal Medicine Group, Department of Obstetrics and Gynaecology, National University of Singapore, Singapore; Pediatric Storage Disorders Laboratory, Institute of Psychiatry, King's College London, London, United Kingdom; University College London (UCL) Institute for Child Health, Gene Transfer Technology Group, Institute for Women's Health, and **Department of Pharmacology, UCL School of Pharmacy, University College London, London, United Kingdom; Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witswatersrand, Johannesburg, South Africa; Department of Reproductive Medicine, KK Women's and Children's Tower, Singapore; and Cancer and Stem Cell Biology, Duke-NUS Graduate Medical School, Singapore.

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http://dx.doi.org/10.1096/fj.14-269092DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4560173PMC
September 2015
78 Reads
2 Citations
5.043 Impact Factor

In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters.

Authors:
Suzanne M K Buckley Juliette M K M Delhove Dany P Perocheau Rajvinder Karda Ahad A Rahim Steven J Howe Natalie J Ward Mark A Birrell Maria G Belvisi Patrick Arbuthnot Mark R Johnson Simon N Waddington Tristan R McKay

Sci Rep 2015 Jul 3;5:11842. Epub 2015 Jul 3.

Stem Cell Group, Cardiovascular &Cell Sciences Research Institute, St. George's University of London, Cranmer Terrace, London SW17 0RE, UK.

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http://dx.doi.org/10.1038/srep11842DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4490336PMC
July 2015
10 Reads
9 Citations
5.080 Impact Factor

The human desmin promoter drives robust gene expression for skeletal muscle stem cell-mediated gene therapy.

Authors:
Jacqueline Jonuschies Michael Antoniou Simon Waddington Luisa Boldrin Francesco Muntoni Adrian Thrasher Jennifer Morgan

Curr Gene Ther 2014 ;14(4):276-88

Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, UCL Institute of Child Health, 30 Guilford Street, London WC1N1EH, UK.

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June 2015
72 Reads
2 Citations
2.542 Impact Factor

Regionally-specified second trimester fetal neural stem cells reveals differential neurogenic programming.

Authors:
Yiping Fan Guillaume Marcy Eddy S M Lee Steve Rozen Citra N Z Mattar Simon N Waddington Eyleen L K Goh Mahesh Choolani Jerry K Y Chan

PLoS One 2014 2;9(9):e105985. Epub 2014 Sep 2.

Department of Reproductive Medicine, KK Women's and Children's Hospital, Singapore, Singapore; Experimental Fetal Medicine Group, Department of Obstetrics and Gynaecology, Yong Loo Lin School of Medicine, National University Health System, Singapore, Singapore; Cancer and Stem Cell Biology Program, Duke-NUS Graduate Medical School, Singapore, Singapore.

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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0105985PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4152177PMC
May 2015
26 Reads
1 Citation
3.234 Impact Factor

Perinatal systemic gene delivery using adeno-associated viral vectors.

Authors:
Rajvinder Karda Suzanne M K Buckley Citra N Mattar Joanne Ng Giulia Massaro Michael P Hughes Manju A Kurian Julien Baruteau Paul Gissen Jerry K Y Chan Chiara Bacchelli Simon N Waddington Ahad A Rahim

Front Mol Neurosci 2014 14;7:89. Epub 2014 Nov 14.

Department of Pharmacology, UCL School of Pharmacy, University College London London, UK.

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http://dx.doi.org/10.3389/fnmol.2014.00089DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4231876PMC
December 2014
22 Reads
3 Citations

Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy.

Authors:
Mario Di Matteo Emira Samara-Kuko Natalie J Ward Simon N Waddington Simon N Waddingon John H McVey Marinee K L Chuah Thierry VandenDriessche

Mol Ther 2014 Sep 18;22(9):1614-24. Epub 2014 Jul 18.

1] Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, Brussels, Belgium [2] Center for Molecular and Vascular Biology, Department of Cardiovascular Medicine, University of Leuven, Leuven, Belgium.

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http://dx.doi.org/10.1038/mt.2014.131DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4435487PMC
September 2014
11 Reads
15 Citations
6.230 Impact Factor

Activator protein 1 is a key terminal mediator of inflammation-induced preterm labor in mice.

Authors:
David A MacIntyre Yun S Lee Roberta Migale Bronwen R Herbert Simon N Waddington Donald Peebles Henrik Hagberg Mark R Johnson Phillip R Bennett

FASEB J 2014 May 4;28(5):2358-68. Epub 2014 Feb 4.

2Imperial College Parturition Research Group, Institute of Reproduction and Developmental Biology, Imperial College London, Hammersmith Campus, London, W12 0NN, UK.

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http://dx.doi.org/10.1096/fj.13-247783DOI Listing
May 2014
9 Reads
23 Citations
5.043 Impact Factor

Transduction of fetal mice with a feline lentiviral vector induces liver tumors which exhibit an E2F activation signature.

Authors:
Reba Condiotti Daniel Goldenberg Hilla Giladi Temima Schnitzer-Perlman Simon N Waddington Suzanne Mk Buckley Denise Heim Wing Cheung Matthew Themis Charles Coutelle Alina Simerzin Emma Osejindu Henning Wege Michael Themis Eithan Galun

Mol Ther 2014 Jan 28;22(1):59-68. Epub 2013 Aug 28.

Goldyne Savad Institute of Gene Therapy, Hadassah Hebrew University Medical Center, Jerusalem, Israel.

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http://dx.doi.org/10.1038/mt.2013.193DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3978808PMC
January 2014
27 Reads
7 Citations
6.230 Impact Factor

Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes.

Authors:
Derek G Burke Ahad A Rahim Simon N Waddington Stefan Karlsson Ida Enquist Kailash Bhatia Atul Mehta Ashok Vellodi Simon Heales

J Inherit Metab Dis 2013 Sep 15;36(5):869-72. Epub 2012 Nov 15.

Clinical & Molecular Genetics Unit, UCL Institute of Child Health and Chemical Pathology, Great Ormond Street Hospital, London, UK.

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http://link.springer.com/10.1007/s10545-012-9561-3
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http://dx.doi.org/10.1007/s10545-012-9561-3DOI Listing
September 2013
26 Reads
6 Citations
3.370 Impact Factor

β-Glucosidase 2 (GBA2) activity and imino sugar pharmacology.

Authors:
Christina M Ridley Karen E Thur Jessica Shanahan Nagendra Babu Thillaiappan Ann Shen Karly Uhl Charlotte M Walden Ahad A Rahim Simon N Waddington Frances M Platt Aarnoud C van der Spoel

J Biol Chem 2013 Sep 23;288(36):26052-66. Epub 2013 Jul 23.

Atlantic Research Centre, Departments of Pediatrics and Biochemistry and Molecular Biology, Dalhousie University, Halifax, Nova Scotia B3H 4R2, Canada.

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http://dx.doi.org/10.1074/jbc.M113.463562DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3764809PMC
September 2013
22 Reads
18 Citations
4.573 Impact Factor

Mitochondria and quality control defects in a mouse model of Gaucher disease--links to Parkinson's disease.

Authors:
Laura D Osellame Ahad A Rahim Iain P Hargreaves Matthew E Gegg Angela Richard-Londt Sebastian Brandner Simon N Waddington Anthony H V Schapira Michael R Duchen

Cell Metab 2013 Jun 23;17(6):941-53. Epub 2013 May 23.

Department of Cell and Developmental Biology, University College London, London WC1E 6BT, UK.

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http://dx.doi.org/10.1016/j.cmet.2013.04.014DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3678026PMC
June 2013
26 Reads
78 Citations
17.570 Impact Factor

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

Authors:
Jenny McIntosh Peter J Lenting Cecilia Rosales Doyoung Lee Samira Rabbanian Deepak Raj Nishil Patel Edward G D Tuddenham Olivier D Christophe John H McVey Simon Waddington Arthur W Nienhuis John T Gray Paolo Fagone Federico Mingozzi Shang-Zhen Zhou Katherine A High Maria Cancio Catherine Y C Ng Junfang Zhou Christopher L Morton Andrew M Davidoff Amit C Nathwani

Blood 2013 Apr 20;121(17):3335-44. Epub 2013 Feb 20.

University College London Cancer Institute, London, United Kingdom.

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http://dx.doi.org/10.1182/blood-2012-10-462200DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3637010PMC
April 2013
49 Reads
60 Citations
10.452 Impact Factor

Exon skipping of hepatic APOB pre-mRNA with splice-switching oligonucleotides reduces LDL cholesterol in vivo.

Authors:
Petra Disterer Raya Al-Shawi Stephan Ellmerich Simon N Waddington James S Owen J Paul Simons Bernard Khoo

Mol Ther 2013 Mar 15;21(3):602-9. Epub 2013 Jan 15.

Institute for Liver and Digestive Health, UCL, London, UK.

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http://linkinghub.elsevier.com/retrieve/pii/S152500161630629
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http://dx.doi.org/10.1038/mt.2012.264DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3589156PMC
March 2013
13 Reads
8 Citations
6.230 Impact Factor

The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis.

Authors:
Ali Nowrouzi Wing T Cheung Tingting Li Xuegong Zhang Anne Arens Anna Paruzynski Simon N Waddington Emma Osejindu Safia Reja Christof von Kalle Yoahe Wang Faisal Al-Allaf Lisa Gregory Matthew Themis Maxine Holder Niraja Dighe Alaine Ruthe Suzanne Mk Buckley Brian Bigger Eugenio Montini Adrian J Thrasher Robert Andrews Terry P Roberts Robert F Newbold Charles Coutelle Manfred Schmidt Mike Themis

Mol Ther 2013 Feb 13;21(2):324-37. Epub 2012 Nov 13.

National Centre for Tumorigenesis, Heidelberg Technology park TP4, Heidelberg, Germany.

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http://dx.doi.org/10.1038/mt.2012.224DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3594025PMC
February 2013
23 Reads
10 Citations
6.230 Impact Factor

The CD46-Jagged1 interaction is critical for human TH1 immunity.

Authors:
Gaëlle Le Friec Devon Sheppard Pat Whiteman Christian M Karsten Salley Al-Tilib Shamoun Adam Laing Laurence Bugeon Margaret J Dallman Teresa Melchionna Chandramouli Chillakuri Richard A Smith Christian Drouet Lionel Couzi Veronique Fremeaux-Bacchi Jörg Köhl Simon N Waddington James M McDonnell Alastair Baker Penny A Handford Susan M Lea Claudia Kemper

Nat Immunol 2012 Dec 21;13(12):1213-21. Epub 2012 Oct 21.

Division of Transplantation Immunology and Mucosal Biology, MRC Centre for Transplantation, King's College London, Guy's Hospital, London, UK.

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http://dx.doi.org/10.1038/ni.2454DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3505834PMC
December 2012
32 Reads
43 Citations
20.004 Impact Factor

The case for intrauterine gene therapy.

Authors:
Citra N Mattar Simon N Waddington Arijit Biswas Andrew M Davidoff Mahesh Choolani Jerry K Y Chan Amit C Nathwani

Best Pract Res Clin Obstet Gynaecol 2012 Oct 20;26(5):697-709. Epub 2012 Jul 20.

Experimental Fetal Medicine Group, Department of Obstetrics and Gynaecology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.

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http://linkinghub.elsevier.com/retrieve/pii/S152169341200104
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http://dx.doi.org/10.1016/j.bpobgyn.2012.06.006DOI Listing
October 2012
14 Reads
4 Citations
3.000 Impact Factor

Vector systems for prenatal gene therapy: choosing vectors for different applications.

Authors:
Charles Coutelle Simon N Waddington

Methods Mol Biol 2012 ;891:41-53

National Heart and Lung Institute, Molecular and Cellular Medicine Section, Imperial College London, London, UK.

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http://dx.doi.org/10.1007/978-1-61779-873-3_3DOI Listing
September 2012
7 Reads

Candidate diseases for prenatal gene therapy.

Authors:
Anna L David Simon N Waddington

Methods Mol Biol 2012 ;891:9-39

Prenatal Cell and Gene Therapy Group, EGA Institute for Women's Health, University College London, London, UK.

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http://dx.doi.org/10.1007/978-1-61779-873-3_2DOI Listing
September 2012
9 Reads
4 Citations

The concept of prenatal gene therapy.

Authors:
Charles Coutelle Simon N Waddington

Methods Mol Biol 2012 ;891:1-7

National Heart and Lung Institute, Molecular and Cellular Medicine Section, Imperial College London, London, UK.

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http://dx.doi.org/10.1007/978-1-61779-873-3_1DOI Listing
September 2012
6 Reads
2 Citations

Monitoring for potential adverse effects of prenatal gene therapy: use of large animal models with relevance to human application.

Authors:
Vedanta Mehta Khalil N Abi-Nader David Carr Jacqueline Wallace Charles Coutelle Simon N Waddington Donald Peebles Anna L David

Methods Mol Biol 2012 ;891:291-328

Prenatal Cell and Gene Therapy Group, EGA Institute for Women's Health, University College London, London, UK.

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http://link.springer.com/10.1007/978-1-61779-873-3_14
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http://dx.doi.org/10.1007/978-1-61779-873-3_14DOI Listing
September 2012
16 Reads
3 Citations

Animal models for prenatal gene therapy: rodent models for prenatal gene therapy.

Authors:
Jessica L Roybal Masayuki Endo Suzanne M K Buckley Bronwen R Herbert Simon N Waddington Alan W Flake

Methods Mol Biol 2012 ;891:201-18

Department of Surgery, Children's Center for Fetal Research and Center for Fetal Diagnosis and Treatment, Children's Hospital of Philadelphia, Philadelphia, PA, USA.

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http://dx.doi.org/10.1007/978-1-61779-873-3_10DOI Listing
September 2012
10 Reads
2 Citations

Monitoring for potential adverse effects of prenatal gene therapy: mouse models for developmental aberrations and inadvertent germ line transmission.

Authors:
Charles Coutelle Simon N Waddington Michael Themis

Methods Mol Biol 2012 ;891:329-40

National Heart and Lung Institute, Molecular and Cellular Medicine Section, Imperial College London, London, UK.

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http://dx.doi.org/10.1007/978-1-61779-873-3_15DOI Listing
September 2012
6 Reads

Perinatal gene transfer to the liver.

Authors:
Tristan R McKay Ahad A Rahim Suzanne M K Buckley Natalie J Ward Jerry K Y Chan Steven J Howe Simon N Waddington

Curr Pharm Des 2011 ;17(24):2528-41

William Harvey Research Institute, Queen Mary University of London, London, UK.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3182410PMC
February 2012
9 Reads
7 Citations
3.452 Impact Factor

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.

Authors:
Christopher Binny Jenny McIntosh Marco Della Peruta Hanna Kymalainen Edward G D Tuddenham Suzanne M K Buckley Simon N Waddington John H McVey Yunyu Spence Christopher L Morton Adrian J Thrasher John T Gray Francis J Castellino Alice F Tarantal Andrew M Davidoff Amit C Nathwani

Blood 2012 Jan 1;119(4):957-66. Epub 2011 Dec 1.

University College London Cancer Institute, London, UK.

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http://dx.doi.org/10.1182/blood-2011-09-377630DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3271720PMC
January 2012
16 Reads
13 Citations
10.452 Impact Factor

Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.

Authors:
Citra N Z Mattar Amit C Nathwani Simon N Waddington Niraja Dighe Christine Kaeppel Ali Nowrouzi Jenny Mcintosh Nuryanti B Johana Bryan Ogden Nicholas M Fisk Andrew M Davidoff Anna David Donald Peebles Marcus B Valentine Jens-Uwe Appelt Christof von Kalle Manfred Schmidt Arijit Biswas Mahesh Choolani Jerry K Y Chan

Mol Ther 2011 Nov 31;19(11):1950-60. Epub 2011 May 31.

Department of Obstetrics and Gynaecology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.

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http://dx.doi.org/10.1038/mt.2011.107DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3222517PMC
November 2011
21 Reads
17 Citations
6.230 Impact Factor

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.

Authors:
Ahad A Rahim Andrew M S Wong Klemens Hoefer Suzanne M K Buckley Citra N Mattar Seng H Cheng Jerry K Y Chan Jonathan D Cooper Simon N Waddington

FASEB J 2011 Oct 11;25(10):3505-18. Epub 2011 Jul 11.

Gene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX, UK.

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http://dx.doi.org/10.1096/fj.11-182311DOI Listing
October 2011
14 Reads
33 Citations
5.043 Impact Factor

Fetal gene therapy: recent advances and current challenges.

Authors:
Citra N Mattar Mahesh Choolani Arijit Biswas Simon N Waddington Jerry K Y Chan

Expert Opin Biol Ther 2011 Oct 31;11(10):1257-71. Epub 2011 May 31.

Yong Loo Lin School of Medicine, National University of Singapore, Department of Obstetrics and Gynaecology, Experimental Fetal Medicine Group, NUHS Tower Block, Level 12, 1E Kent Ridge Road, 119228 Singapore.

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http://dx.doi.org/10.1517/14712598.2011.585153DOI Listing
October 2011
18 Reads
5 Citations
3.743 Impact Factor

Organ targeted prenatal gene therapy--how far are we?

Authors:
Vedanta Mehta Khalil Abi Nader Simon Waddington Anna L David

Prenat Diagn 2011 Jul 25;31(7):720-34. Epub 2011 May 25.

Prenatal Cell and Gene Therapy Group, Institute for Women's Health, University College London, London, UK.

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http://dx.doi.org/10.1002/pd.2787DOI Listing
July 2011
8 Reads
3 Citations
2.514 Impact Factor

Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver.

Authors:
Orestis Argyros Suet Ping Wong Constantinos Fedonidis Oleg Tolmachov Simon N Waddington Steven J Howe Marcello Niceta Charles Coutelle Richard P Harbottle

J Mol Med (Berl) 2011 May 8;89(5):515-29. Epub 2011 Feb 8.

Gene Therapy Research Group, Section of Molecular Medicine, National Heart and Lung Institute, Imperial College London, London, UK.

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http://dx.doi.org/10.1007/s00109-010-0713-3DOI Listing
May 2011
20 Reads
13 Citations
5.110 Impact Factor

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.

Authors:
Amit C Nathwani Cecilia Rosales Jenny McIntosh Ghasem Rastegarlari Devhrut Nathwani Deepak Raj Sushmita Nawathe Simon N Waddington Roderick Bronson Scott Jackson Robert E Donahue Katherine A High Federico Mingozzi Catherine Y C Ng Junfang Zhou Yunyu Spence M Beth McCarville Marc Valentine James Allay John Coleman Susan Sleep John T Gray Arthur W Nienhuis Andrew M Davidoff

Mol Ther 2011 May 18;19(5):876-85. Epub 2011 Jan 18.

Department of Hematology, University College London Cancer Institute, London, UK.

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http://dx.doi.org/10.1038/mt.2010.274DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3098629PMC
May 2011
53 Reads
89 Citations
6.230 Impact Factor

Perinatal gene delivery to the CNS.

Authors:
Ahad A Rahim Suzanne M K Buckley Jerry K Y Chan Donald M Peebles Simon N Waddington

Ther Deliv 2011 Apr;2(4):483-91

Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WCIE 6HX, UK.

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April 2011
7 Reads
1 Citation

Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep.

Authors:
Anna L David Jenny McIntosh Donald M Peebles Terry Cook Simon Waddington Boaz Weisz Victoria Wigley Khalil Abi-Nader Michael Boyd Andrew M Davidoff Amit C Nathwani

Hum Gene Ther 2011 Apr 2;22(4):419-26. Epub 2011 Feb 2.

Institute for Women's Health, University College London, London WC1E 6HX, United Kingdom.

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http://dx.doi.org/10.1089/hum.2010.007DOI Listing
April 2011
20 Reads
17 Citations
3.755 Impact Factor

Codon optimization of human factor VIII cDNAs leads to high-level expression.

Authors:
Natalie J Ward Suzanne M K Buckley Simon N Waddington Thierry Vandendriessche Marinee K L Chuah Amit C Nathwani Jenny McIntosh Edward G D Tuddenham Christine Kinnon Adrian J Thrasher John H McVey

Blood 2011 Jan 1;117(3):798-807. Epub 2010 Nov 1.

Molecular Immunology Unit, Institute of Child Health, University College London, London, UK.

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http://www.bloodjournal.org/cgi/doi/10.1182/blood-2010-05-28
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http://dx.doi.org/10.1182/blood-2010-05-282707DOI Listing
January 2011
14 Reads
51 Citations
10.452 Impact Factor

In utero gene transfer to the mouse nervous system.

Authors:
Ahad A Rahim Andrew M S Wong Suzanne M K Buckley Jerry K Y Chan Anna L David Jonathan D Cooper Charles Coutelle Donald M Peebles Simon N Waddington

Biochem Soc Trans 2010 Dec;38(6):1489-93

Institute for Women's Health, University College London, 86-96 Chenies Mews, London WC1E 6HX, UK.

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http://dx.doi.org/10.1042/BST0381489DOI Listing
December 2010
7 Reads
3 Citations
3.194 Impact Factor

Gene delivery of a mutant TGFβ3 reduces markers of scar tissue formation after cutaneous wounding.

Authors:
Simon N Waddington Rachel Crossley Vicky Sheard Steven J Howe Suzanne M K Buckley Lynda Coughlan David E Gilham Robert E Hawkins Tristan R McKay

Mol Ther 2010 Dec 24;18(12):2104-11. Epub 2010 Aug 24.

Institute for Women's Health, University College London, London, UK.

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http://dx.doi.org/10.1038/mt.2010.174DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2997585PMC
December 2010
14 Reads
9 Citations
6.230 Impact Factor

Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis.

Authors:
Andrew M S Wong Ahad A Rahim Simon N Waddington Jonathan D Cooper

Biochem Soc Trans 2010 Dec;38(6):1484-8

Pediatric Storage Disorders Laboratory, Department of Neuroscience and Centre for the Cellular Basis of Behaviour, MRC Centre for Neurodegeneration Research, James Black Centre, Institute of Psychiatry, King's College London, 125 Coldharbour Lane, London SE5 9NU, UK.

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December 2010
11 Reads
11 Citations
3.194 Impact Factor

LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives.

Authors:
Faisal A Al-Allaf Charles Coutelle Simon N Waddington Anna L David Richard Harbottle Michael Themis

Int Arch Med 2010 Dec 13;3:36. Epub 2010 Dec 13.

Department of Medical Genetics, Faculty of Medicine, Umm Al-Qura University, Al-Abedia Campus, P, O, Box 715, Makkah 21955, Saudi Arabia.

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http://dx.doi.org/10.1186/1755-7682-3-36DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3016243PMC
December 2010
9 Reads
11 Citations

Functional characterization of a 13-bp deletion (c.-1522_-1510del13) in the promoter of the von Willebrand factor gene in type 1 von Willebrand disease.

Authors:
Maha Othman Yvette Chirinian Christine Brown Colleen Notley Nicholas Hickson Daniel Hampshire Suzanne Buckley Simon Waddington Alan L Parker Andrew Baker Paula James David Lillicrap

Blood 2010 Nov 9;116(18):3645-52. Epub 2010 Aug 9.

Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada.

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http://dx.doi.org/10.1182/blood-2009-12-261131DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2981484PMC
November 2010
12 Reads
5 Citations
10.452 Impact Factor

Biodistribution and retargeting of FX-binding ablated adenovirus serotype 5 vectors.

Authors:
Raul Alba Angela C Bradshaw Lynda Coughlan Laura Denby Robert A McDonald Simon N Waddington Suzanne M K Buckley Jenny A Greig Alan L Parker Ashley M Miller Hongjie Wang Andre Lieber Nico van Rooijen John H McVey Stuart A Nicklin Andrew H Baker

Blood 2010 Oct 7;116(15):2656-64. Epub 2010 Jul 7.

British Heart Foundation Glasgow Cardiovascular Research Centre, University of Glasgow, Glasgow, UK.

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http://dx.doi.org/10.1182/blood-2009-12-260026DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2974579PMC
October 2010
14 Reads
40 Citations
10.452 Impact Factor

Neonatal gene therapy of glycogen storage disease type Ia using a feline immunodeficiency virus-based vector.

Authors:
Albert Grinshpun Reba Condiotti Simon N Waddington Michael Peer Eli Zeig Sima Peretz Alina Simerzin Janice Chou Chi-Jiunn Pann Hilla Giladi Eithan Galun

Mol Ther 2010 Sep 22;18(9):1592-8. Epub 2010 Jun 22.

Goldyne Savad Institute of Gene Therapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

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http://dx.doi.org/10.1038/mt.2010.119DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2956916PMC
September 2010
12 Reads
10 Citations
6.230 Impact Factor

Increased secretion of lipoproteins in transgenic mice expressing human D374Y PCSK9 under physiological genetic control.

Authors:
Bronwen Herbert Dilipkumar Patel Simon N Waddington Emily R Eden Alexandra McAleenan Xi-Ming Sun Anne K Soutar

Arterioscler Thromb Vasc Biol 2010 Jul 6;30(7):1333-9. Epub 2010 May 6.

Medical Research Council Clinical Sciences Centre, Imperial College London, Hammersmith Hospital, Ducane Road, London W12 0NN, England.

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http://dx.doi.org/10.1161/ATVBAHA.110.204040DOI Listing
July 2010
10 Reads
13 Citations
6.000 Impact Factor

Desmin-regulated lentiviral vectors for skeletal muscle gene transfer.

Authors:
Gillian E Talbot Simon N Waddington Olivia Bales Rose C Tchen Michael N Antoniou

Mol Ther 2010 Mar 24;18(3):601-8. Epub 2009 Nov 24.

King's College London School of Medicine, Nuclear Biology Group, Department of Medical and Molecular Genetics, Guy's Hospital, London, UK.

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http://dx.doi.org/10.1038/mt.2009.267DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2839432PMC
March 2010
11 Reads
10 Citations
6.230 Impact Factor

Activation and deactivation of periventricular white matter phagocytes during postnatal mouse development.

Authors:
Mariya Hristova Daniel Cuthill Virginia Zbarsky Alejandro Acosta-Saltos Adam Wallace Ken Blight Suzanne M K Buckley Donald Peebles Heike Heuer Simon N Waddington Gennadij Raivich

Glia 2010 Jan;58(1):11-28

Department of Obstetrics and Gynecology, EGA Institute of Women's Health, University College London, London, United Kingdom.

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http://dx.doi.org/10.1002/glia.20896DOI Listing
January 2010
10 Reads
28 Citations
6.031 Impact Factor

The differentiation and engraftment potential of mouse hematopoietic stem cells is maintained after bio-electrospray.

Authors:
Kerol Bartolovic Napachanok Mongkoldhumrongkul Simon N Waddington Suwan N Jayasinghe Steven J Howe

Analyst 2010 Jan 13;135(1):157-64. Epub 2009 Nov 13.

Wolfson Centre for Gene Therapy, Molecular Immunology Unit, UCL Institute of Child Health, University College London, 30 Guilford Street, London, UK.

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http://dx.doi.org/10.1039/b917813aDOI Listing
January 2010
7 Reads
2 Citations
4.110 Impact Factor

Influence of coagulation factor x on in vitro and in vivo gene delivery by adenovirus (Ad) 5, Ad35, and chimeric Ad5/Ad35 vectors.

Authors:
Jenny A Greig Suzanne Mk Buckley Simon N Waddington Alan L Parker David Bhella Rebecca Pink Ahad A Rahim Takashi Morita Stuart A Nicklin John H McVey Andrew H Baker

Mol Ther 2009 Oct 14;17(10):1683-91. Epub 2009 Jul 14.

British Heart Foundation Glasgow Cardiovascular Research Centre, University of Glasgow, Glasgow G12 8TA, UK.

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http://dx.doi.org/10.1038/mt.2009.152DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2835023PMC
October 2009
15 Reads
18 Citations
6.230 Impact Factor

Identification of coagulation factor (F)X binding sites on the adenovirus serotype 5 hexon: effect of mutagenesis on FX interactions and gene transfer.

Authors:
Raul Alba Angela C Bradshaw Alan L Parker David Bhella Simon N Waddington Stuart A Nicklin Nico van Rooijen Jerome Custers Jaap Goudsmit Dan H Barouch John H McVey Andrew H Baker

Blood 2009 Jul 8;114(5):965-71. Epub 2009 May 8.

British Heart Foundation Glasgow Cardiovascular Research Centre, University of Glasgow, G12 8TA, UK.

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http://dx.doi.org/10.1182/blood-2009-03-208835DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2721791PMC
July 2009
12 Reads
70 Citations
10.452 Impact Factor

Differentiation of human fetal mesenchymal stem cells into cells with an oligodendrocyte phenotype.

Authors:
Nigel L Kennea Simon N Waddington Jerry Chan Keelin O'Donoghue Davy Yeung Deanna L Taylor Faisal A Al-Allaf Grisha Pirianov Michael Themis A David Edwards Nicholas M Fisk Huseyin Mehmet

Cell Cycle 2009 Apr 9;8(7):1069-79. Epub 2009 Apr 9.

Institute of Reproductive and Developmental Biology, Faculty of Medicine, Imperial College London, London, UK.

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http://dx.doi.org/10.4161/cc.8.7.8121DOI Listing
April 2009
10 Reads
17 Citations

The cyclopentenone 15-deoxy-delta 12,14-prostaglandin J(2) delays lipopolysaccharide-induced preterm delivery and reduces mortality in the newborn mouse.

Authors:
Grisha Pirianov Simon N Waddington Tamsin M Lindström Vasiliki Terzidou Huseyin Mehmet Phillip R Bennett

Endocrinology 2009 Feb 9;150(2):699-706. Epub 2008 Oct 9.

Imperial College Parturition Research Group, Department of Reproductive Biology, Institute of Reproductive and Developmental Biology, Imperial College London, Hammersmith Campus, London, UK.

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http://dx.doi.org/10.1210/en.2008-1178DOI Listing
February 2009
12 Reads
27 Citations
4.503 Impact Factor

Effect of neutralizing sera on factor x-mediated adenovirus serotype 5 gene transfer.

Authors:
Alan L Parker Simon N Waddington Suzanne M K Buckley Jerome Custers Menzo J E Havenga Nico van Rooijen Jaap Goudsmit John H McVey Stuart A Nicklin Andrew H Baker

J Virol 2009 Jan 22;83(1):479-83. Epub 2008 Oct 22.

British Heart Foundation Glasgow Cardiovascular Research Centre, Division of Cardiovascular and Medical Sciences, 126 University Place, University of Glasgow, Glasgow G12 8TA, United Kingdom.

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http://dx.doi.org/10.1128/JVI.01878-08DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2612319PMC
January 2009
50 Reads
26 Citations
4.440 Impact Factor

Luciferin detection after intranasal vector delivery is improved by intranasal rather than intraperitoneal luciferin administration.

Authors:
Suzanne M K Buckley Steven J Howe Ahad A Rahim Hildegard Buning Jenny McIntosh Suet-Ping Wong Andrew H Baker Amit Nathwani Adrian J Thrasher Charles Coutelle Tristan R McKay Simon N Waddington

Hum Gene Ther 2008 Oct;19(10):1050-6

Department of Haematology, Haemophilia Centre and Haemostasis Unit, Royal Free and University College Medical School, London NW3 2PF, UK.

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http://dx.doi.org/10.1089/hum.2008.023DOI Listing
October 2008
28 Reads
14 Citations
3.755 Impact Factor

Adenovirus serotype 5 hexon mediates liver gene transfer.

Authors:
Simon N Waddington John H McVey David Bhella Alan L Parker Kristeen Barker Hideko Atoda Rebecca Pink Suzanne M K Buckley Jenny A Greig Laura Denby Jerome Custers Takashi Morita Ivo M B Francischetti Robson Q Monteiro Dan H Barouch Nico van Rooijen Claudio Napoli Menzo J E Havenga Stuart A Nicklin Andrew H Baker

Cell 2008 Feb;132(3):397-409

Department of Haematology, Haemophilia Centre and Haemostasis Unit, Royal Free and University College Medical School, London NW3 2PF, UK.

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http://dx.doi.org/10.1016/j.cell.2008.01.016DOI Listing
February 2008
14 Reads
241 Citations
32.242 Impact Factor

Stable gene transfer to muscle using non-integrating lentiviral vectors.

Authors:
Luis Apolonia Simon N Waddington Carolina Fernandes Natalie J Ward Gerben Bouma Michael P Blundell Adrian J Thrasher Mary K Collins Nicola J Philpott

Mol Ther 2007 Nov 14;15(11):1947-54. Epub 2007 Aug 14.

Molecular Immunology Unit, Institute of Child Health, London, UK.

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http://dx.doi.org/10.1038/sj.mt.6300281DOI Listing
November 2007
14 Reads
75 Citations
6.230 Impact Factor

Fetal gene transfer.

Authors:
Simon N Waddington Suzanne Mk Buckley Anna L David Donald M Peebles Charles H Rodeck Charles Coutelle

Curr Opin Mol Ther 2007 Oct;9(5):432-8

University College London, Department of Haematology, Haemophilia Centre and Haemostasis Unit, Royal Free and University College London Medical School, Rowland Hill Street, London, NW3 2PF, UK.

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October 2007
11 Reads
6 Citations

Targeting of adenovirus serotype 5 (Ad5) and 5/47 pseudotyped vectors in vivo: fundamental involvement of coagulation factors and redundancy of CAR binding by Ad5.

Authors:
Simon N Waddington Alan L Parker Menzo Havenga Stuart A Nicklin Suzanne M K Buckley John H McVey Andrew H Baker

J Virol 2007 Sep 6;81(17):9568-71. Epub 2007 Jun 6.

Department of Haematology, Haemophilia Centre and Haemostasis Unit, Royal Free and University College Medical School, London NW3 2PF, United Kingdom.

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http://dx.doi.org/10.1128/JVI.00663-07DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1951445PMC
September 2007
9 Reads
34 Citations
4.440 Impact Factor

The influence of blood on in vivo adenovirus bio-distribution and transduction.

Authors:
Andrew H Baker John H Mcvey Simon N Waddington Nelson C Di Paolo Dmitry M Shayakhmetov

Mol Ther 2007 Aug 15;15(8):1410-6. Epub 2007 May 15.

British Heart Foundation Glasgow Cardiovascular Research Centre, University of Glasgow, Glasgow, UK.

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http://dx.doi.org/10.1038/sj.mt.6300206DOI Listing
August 2007
11 Reads
24 Citations
6.230 Impact Factor

Delivery and long-term expression of a 135 kb LDLR genomic DNA locus in vivo by hydrodynamic tail vein injection.

Authors:
Olivia C Hibbitt Richard P Harbottle Simon N Waddington Christine A Bursill Charles Coutelle Keith M Channon Richard Wade-Martins

J Gene Med 2007 Jun;9(6):488-97

Wellcome Trust Centre for Human Genetics, University of Oxford, Roosevelt Drive, Oxford, UK.

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http://doi.wiley.com/10.1002/jgm.1041
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http://dx.doi.org/10.1002/jgm.1041DOI Listing
June 2007
10 Reads
12 Citations
2.472 Impact Factor

Widespread distribution and muscle differentiation of human fetal mesenchymal stem cells after intrauterine transplantation in dystrophic mdx mouse.

Authors:
Jerry Chan Simon N Waddington Keelin O'Donoghue Hitoshi Kurata Pascale V Guillot Cecilia Gotherstrom Michael Themis Jennifer E Morgan Nicholas M Fisk

Stem Cells 2007 Apr 21;25(4):875-84. Epub 2006 Dec 21.

Experimental Fetal Medicine Group, Institute of Reproductive and Developmental Biology, Imperial College London, Hammersmith Campus, Du Cane Road, London, United Kingdom.

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http://doi.wiley.com/10.1634/stemcells.2006-0694
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April 2007
11 Reads
32 Citations
6.523 Impact Factor

Influence of coagulation factor zymogens on the infectivity of adenoviruses pseudotyped with fibers from subgroup D.

Authors:
Alan L Parker John H McVey Jessica H Doctor Oscar Lopez-Franco Simon N Waddington Menzo J E Havenga Stuart A Nicklin Andrew H Baker

J Virol 2007 Apr 24;81(7):3627-31. Epub 2007 Jan 24.

British Heart Foundation Glasgow Cardiovascular Research Centre, Division of Cardiovascular and Medical Sciences, University of Glasgow, 126 University Place, Glasgow G12 8TA, United Kingdom.

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http://jvi.asm.org/cgi/doi/10.1128/JVI.02786-06
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http://dx.doi.org/10.1128/JVI.02786-06DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1866077PMC
April 2007
38 Reads
23 Citations
4.440 Impact Factor

Multiple vitamin K-dependent coagulation zymogens promote adenovirus-mediated gene delivery to hepatocytes.

Authors:
Alan L Parker Simon N Waddington Campbell G Nicol Dmitry M Shayakhmetov Suzanne M Buckley Laura Denby Geoffrey Kemball-Cook Shaoheng Ni Andre Lieber John H McVey Stuart A Nicklin Andrew H Baker

Blood 2006 Oct 20;108(8):2554-61. Epub 2006 Jun 20.

British Heart Foundation Glasgow Cardiovascular Research Centre, Division of Cardiovascular and Medical Sciences, 126 University Place, University of Glasgow, G12 8TA, United Kingdom.

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http://dx.doi.org/10.1182/blood-2006-04-008532DOI Listing
October 2006
14 Reads
116 Citations
10.452 Impact Factor

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.

Authors:
Amit C Nathwani John T Gray Catherine Y C Ng Junfang Zhou Yunyu Spence Simon N Waddington Edward G D Tuddenham Geoffrey Kemball-Cook Jenny McIntosh Mariette Boon-Spijker Koen Mertens Andrew M Davidoff

Blood 2006 Apr 1;107(7):2653-61. Epub 2005 Dec 1.

Department of Haematology, University College London, 98 Chenies Mews, London, United Kingdom, WC1E 6HX.

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http://dx.doi.org/10.1182/blood-2005-10-4035DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1895379PMC
April 2006
14 Reads
125 Citations
10.452 Impact Factor

Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice.

Authors:
Mike Themis Simon N Waddington Manfred Schmidt Christof von Kalle Yoahe Wang Faisal Al-Allaf Lisa G Gregory Megha Nivsarkar Matthew Themis Maxine V Holder Suzanne M K Buckley Niraja Dighe Alaine T Ruthe Ajay Mistry Brian Bigger Ahad Rahim Tuan H Nguyen Didier Trono Adrian J Thrasher Charles Coutelle

Mol Ther 2005 Oct;12(4):763-71

Gene Therapy Research Group, Section of Cell and Molecular Biology, Imperial College London, UK.

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http://dx.doi.org/10.1016/j.ymthe.2005.07.358DOI Listing
October 2005
18 Reads
51 Citations
6.230 Impact Factor

Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy.

Authors:
Boaz Weisz Anna L David Lisa G Gregory Dany Perocheau Ali Ruthe Simon N Waddington Mike Themis Terry Cook Charles Coutelle Charles H Rodeck Donald M Peebles

Am J Obstet Gynecol 2005 Sep;193(3 Pt 2):1105-9

Department of Obstetrics and Gynaecology, University College London, United Kingdom.

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http://dx.doi.org/10.1016/j.ajog.2005.06.077DOI Listing
September 2005
29 Reads
4.704 Impact Factor

No evidence for germ-line transmission following prenatal and early postnatal AAV-mediated gene delivery.

Authors:
Marcus Jakob Christiane Mühle Jung Park Susi Weiss Simon Waddington Holm Schneider

J Gene Med 2005 May;7(5):630-7

Department of Experimental Medicine I, Nikolaus Fiebiger Centre of Molecular Medicine, University of Erlangen-Nuernberg, Germany.

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http://dx.doi.org/10.1002/jgm.718DOI Listing
May 2005
11 Reads
5 Citations
2.472 Impact Factor

In utero gene therapy: current challenges and perspectives.

Authors:
Simon N Waddington M Gabriela Kramer Ruben Hernandez-Alcoceba Suzanne M K Buckley Michael Themis Charles Coutelle Jesus Prieto

Mol Ther 2005 May;11(5):661-76

Gene Therapy Research Group, Sir Alexander Fleming Building, Imperial College, South Kensington, London SW7 2AZ, UK.

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http://dx.doi.org/10.1016/j.ymthe.2005.01.015DOI Listing
May 2005
7 Reads
10 Citations
6.230 Impact Factor

Complement inhibition rescued mice allowing observation of transgene expression following intraportal delivery of baculovirus in mice.

Authors:
Jonathan Hoare Simon Waddington Howard C Thomas Charles Coutelle Michael J McGarvey

J Gene Med 2005 Mar;7(3):325-33

Division of Medicine, Faculty of Medicine, Imperial College London, St Mary's Campus, South Wharf Road, London W2 1NY, UK.

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http://dx.doi.org/10.1002/jgm.671DOI Listing
March 2005
9 Reads
9 Citations
2.472 Impact Factor

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.

Authors:
Simon N Waddington Megha S Nivsarkar Ajay R Mistry Suzanne M K Buckley Geoffrey Kemball-Cook Karen L Mosley Kyriacos Mitrophanous Pippa Radcliffe Maxine V Holder Mairi Brittan Anastasios Georgiadis Faisal Al-Allaf Brian W Bigger Lisa G Gregory H Terence Cook Robin R Ali Adrian Thrasher Edward G D Tuddenham Mike Themis Charles Coutelle

Blood 2004 Nov 1;104(9):2714-21. Epub 2004 Jul 1.

Imperial College London, Gene Therapy Research Group, Section of Cell and Molecular Biology, Sir Alexander Fleming Bldg, Imperial College Road, London, SW7 2AZ, United Kingdom.

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http://dx.doi.org/10.1182/blood-2004-02-0627DOI Listing
November 2004
19 Reads
29 Citations
10.452 Impact Factor

Ultrasound-guided percutaneous delivery of adenoviral vectors encoding the beta-galactosidase and human factor IX genes to early gestation fetal sheep in utero.

Authors:
Anna David Terry Cook Simon Waddington Donald Peebles Megha Nivsarkar Holly Knapton Maznu Miah Thomas Dahse David Noakes Holm Schneider Charles Rodeck Charles Coutelle Mike Themis

Hum Gene Ther 2003 Mar;14(4):353-64

Department of Obstetrics and Gynaecology, Royal Free and University College Medical School, 86-96 Chenies Mews, London, WC1E 6HX, United Kingdom.

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http://dx.doi.org/10.1089/104303403321208952DOI Listing
March 2003
57 Reads
8 Citations
3.755 Impact Factor

In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor.

Authors:
Simon N Waddington Suzanne M K Buckley Megha Nivsarkar Sarah Jezzard Holm Schneider Thomas Dahse Geoff Kemball-Cook Maznu Miah Nick Tucker Margaret J Dallman Mike Themis Charles Coutelle

Blood 2003 Feb 3;101(4):1359-66. Epub 2002 Oct 3.

Gene Therapy, Section of Cell and Molecular Biology, Imperial College School of Science, Technology and Medicine, London, United Kingdom.

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http://dx.doi.org/10.1182/blood-2002-03-0779DOI Listing
February 2003
19 Reads
22 Citations
10.452 Impact Factor

Sustained delivery of therapeutic concentrations of human clotting factor IX--a comparison of adenoviral and AAV vectors administered in utero.

Authors:
Holm Schneider Christiane Mühle Anne Marie Douar Simon Waddington Qiu-Jie Jiang Klaus von der Mark Charles Coutelle Wolfgang Rascher

J Gene Med 2002 Jan-Feb;4(1):46-53

Children's Hospital, University of Erlangen-Nuernberg, Germany.

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April 2002
43 Reads
10 Citations
2.472 Impact Factor

Arginase in glomerulonephritis.

Authors:
Simon N Waddington

Kidney Int 2002 Mar;61(3):876-81

Gene Therapy Group, Section of Cell and Molecular Biology, Faculty of Medicine, Imperial College School of Science Technology and Medicine, Sir Alexander Fleming Building, Imperial College Road, London, England SW7 2AZ, UK.

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http://dx.doi.org/10.1046/j.1523-1755.2002.00236.xDOI Listing
March 2002
7 Reads
3 Citations
8.563 Impact Factor

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Charles Coutelle
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National Heart and Lung Institute

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Ahad A Rahim
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UCL School of Pharmacy

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John H McVey
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Alan L Parker
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CRC Institute for Cancer Studies

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Andrew H Baker
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Institute of Cardiovascular and Medical Sciences

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Steven J Howe

UCL Institute of Child Health

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University College London Cancer Institute

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