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:

Metrics

Number of Publications

82

Publications

Number of Profile Views

589

Profile Views

Number of Article Reads

1076

Reads

Number of Citations

1755

Citations

Education

Jan 1999
Imperial College London
PhD

Top co-authors

Charles Coutelle
Charles Coutelle

National Heart and Lung Institute

20
Ahad A Rahim
Ahad A Rahim

UCL School of Pharmacy

15
Alan L Parker
Alan L Parker

CRC Institute for Cancer Studies

10
Andrew H Baker
Andrew H Baker

Institute of Cardiovascular and Medical Sciences

10
Anna L David
Anna L David

Institute for Women's Health

8
Steven J Howe
Steven J Howe

UCL Institute of Child Health

8
Amit C Nathwani
Amit C Nathwani

University College London Cancer Institute

8

Publications

82Publications

1076Reads

1755PubMed Central Citations

Vps33b is crucial for structural and functional hepatocyte polarity.

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:

View Article
May 2017
12 Reads
3 Citations
11.340 Impact Factor

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

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

View Article
December 2016
7 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.

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.

View Article
November 2016
20 Reads
2.540 Impact Factor

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

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.

View Article
June 2016
16 Reads
3 Citations
7.250 Impact Factor

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

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:

View Article
March 2016
38 Reads
21 Citations
7.210 Impact Factor

Gene Therapy with Adeno-associated Virus for Cystic Fibrosis.

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

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

View Article
February 2016
10 Reads
1 Citation
13.000 Impact Factor

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

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.

View Article
February 2016
9 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.

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.

View Article
December 2015
25 Reads
4 Citations

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

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.

View Article
October 2015
13 Reads
2 Citations
3.170 Impact Factor

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

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:

View Article
September 2015
11 Reads
7 Citations
4.590 Impact Factor

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

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.

View Article
September 2015
42 Reads
2 Citations
5.040 Impact Factor

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

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.

View Article
July 2015
7 Reads
9 Citations
5.080 Impact Factor

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

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.

View Article
June 2015
43 Reads
2 Citations
2.540 Impact Factor

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

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.

View Article
May 2015
19 Reads
1 Citation
3.230 Impact Factor

Perinatal systemic gene delivery using adeno-associated viral vectors.

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

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

View Article
December 2014
19 Reads
3 Citations

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

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.

View Article
September 2014
10 Reads
15 Citations
6.230 Impact Factor

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

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.

View Article
May 2014
8 Reads
23 Citations
5.040 Impact Factor

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

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.

View Article
September 2013
18 Reads
6 Citations
3.370 Impact Factor

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

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.

View Article
September 2013
19 Reads
18 Citations
4.570 Impact Factor

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

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.

View Article
June 2013
15 Reads
78 Citations
17.570 Impact Factor

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

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

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

View Article
March 2013
8 Reads
8 Citations
6.230 Impact Factor

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

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.

View Article
December 2012
16 Reads
43 Citations
20.000 Impact Factor

The case for intrauterine gene therapy.

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.

View Article
October 2012
11 Reads
4 Citations
3.000 Impact Factor

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

Methods Mol Biol 2012 ;891:41-53

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

View Article
September 2012
7 Reads

Choice of surrogate and physiological markers for prenatal gene therapy.

Methods Mol Biol 2012 ;891:273-90

Gene Transfer Technology Group, Institute for Women's Health, University College London, London, UK.

View Article
September 2012
3 Reads

Candidate diseases for prenatal gene therapy.

Methods Mol Biol 2012 ;891:9-39

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

View Article
September 2012
8 Reads
4 Citations

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

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.

View Article
September 2012
8 Reads
2 Citations

The concept of prenatal gene therapy.

Methods Mol Biol 2012 ;891:1-7

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

View Article
September 2012
5 Reads
2 Citations

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

Methods Mol Biol 2012 ;891:291-328

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

View Article
September 2012
14 Reads
3 Citations

Perinatal gene transfer to the liver.

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

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

View Article
February 2012
6 Reads
7 Citations
3.450 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.

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.

View Article
October 2011
9 Reads
33 Citations
5.040 Impact Factor

Fetal gene therapy: recent advances and current challenges.

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.

View Article
October 2011
9 Reads
5 Citations
3.740 Impact Factor

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

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.

View Article
July 2011
6 Reads
3 Citations
2.510 Impact Factor

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

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.

View Article
May 2011
15 Reads
13 Citations
5.110 Impact Factor

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

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.

View Article
April 2011
18 Reads
17 Citations
3.750 Impact Factor

Perinatal gene delivery to the CNS.

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

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

View Article
April 2011
6 Reads
1 Citation

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

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

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

View Article
January 2011
9 Reads
51 Citations
10.450 Impact Factor

In utero gene transfer to the mouse nervous system.

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.

View Article
December 2010
5 Reads
3 Citations
3.190 Impact Factor

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

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

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

View Article
December 2010
8 Reads
9 Citations
6.230 Impact Factor

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

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.

View Article
December 2010
9 Reads
11 Citations
3.190 Impact Factor

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

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.

View Article
December 2010
6 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.

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

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

View Article
November 2010
10 Reads
5 Citations
10.450 Impact Factor

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

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

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

View Article
October 2010
9 Reads
40 Citations
10.450 Impact Factor

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

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.

View Article
September 2010
7 Reads
10 Citations
6.230 Impact Factor

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

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.

View Article
July 2010
10 Reads
13 Citations
6.000 Impact Factor

Desmin-regulated lentiviral vectors for skeletal muscle gene transfer.

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.

View Article
March 2010
8 Reads
10 Citations
6.230 Impact Factor

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

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.

View Article
January 2010
6 Reads
2 Citations
4.110 Impact Factor

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

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

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

View Article
January 2010
9 Reads
28 Citations
6.030 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.

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.

View Article
October 2009
8 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.

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.

View Article
July 2009
6 Reads
70 Citations
10.450 Impact Factor

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

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.

View Article
April 2009
8 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.

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.

View Article
February 2009
11 Reads
27 Citations
4.500 Impact Factor

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

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.

View Article
January 2009
34 Reads
26 Citations
4.440 Impact Factor

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

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.

View Article
October 2008
23 Reads
14 Citations
3.750 Impact Factor

Adenovirus serotype 5 hexon mediates liver gene transfer.

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.

View Article
February 2008
10 Reads
241 Citations
32.240 Impact Factor

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

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

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

View Article
November 2007
9 Reads
75 Citations
6.230 Impact Factor

Fetal gene transfer.

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.

View Article
October 2007
10 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.

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.

View Article
September 2007
7 Reads
34 Citations
4.440 Impact Factor

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

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.

View Article
August 2007
9 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.

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

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

View Article
June 2007
8 Reads
12 Citations
2.470 Impact Factor

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

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.

View Article
April 2007
15 Reads
23 Citations
4.440 Impact Factor

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

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.

View Article
April 2007
11 Reads
32 Citations
6.520 Impact Factor

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

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.

View Article
October 2006
12 Reads
116 Citations
10.450 Impact Factor

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

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

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

View Article
September 2005
18 Reads
4.700 Impact Factor

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

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.

View Article
May 2005
10 Reads
5 Citations
2.470 Impact Factor

In utero gene therapy: current challenges and perspectives.

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

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

View Article
May 2005
5 Reads
10 Citations
6.230 Impact Factor

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

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.

View Article
March 2005
7 Reads
9 Citations
2.470 Impact Factor

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

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.

View Article
November 2004
12 Reads
29 Citations
10.450 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.

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.

View Article
March 2003
34 Reads
8 Citations
3.750 Impact Factor

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

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.

View Article
February 2003
15 Reads
22 Citations
10.450 Impact Factor

Arginase in glomerulonephritis.

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.

View Article
March 2002
6 Reads
3 Citations
8.560 Impact Factor

Top co-authors

Charles Coutelle
Charles Coutelle

National Heart and Lung Institute

20
Ahad A Rahim
Ahad A Rahim

UCL School of Pharmacy

15
Alan L Parker
Alan L Parker

CRC Institute for Cancer Studies

10
Andrew H Baker
Andrew H Baker

Institute of Cardiovascular and Medical Sciences

10
Anna L David
Anna L David

Institute for Women's Health

8
Steven J Howe
Steven J Howe

UCL Institute of Child Health

8
Amit C Nathwani
Amit C Nathwani

University College London Cancer Institute

8