All Stories

  1. Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector–mediated intrauterine gene transfer in early-gestation fetal macaques

    Article • The FASEB Journal, December 2018, Federation of American Societies For Experimental Biology (FASEB)

    Dr Simon N. Waddington

  2. Vps33b is crucial for structural and functional hepatocyte polarity

    Article • Journal of Hepatology, May 2017, Elsevier

    Dr Simon N. Waddington

  3. Lentiviral vectors can be used for full-length dystrophin gene therapy

    Article • Scientific Reports, March 2017, Nature

    Dr Simon N. Waddington

  4. Delivering efficient liver-directed AAV-mediated gene therapy

    Article • Gene Therapy, January 2017, Nature

    Dr Simon N. Waddington

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

    Article • Biology of Reproduction, October 2016, Society for the Study of Reproduction

    Dr Simon N. Waddington

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

    Article • Nature Communications, July 2016, Nature

    Dr Janos KristonVizi, Dr Simon N. Waddington

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

    Article • BMC Medicine, June 2016, Springer Science + Business Media

    Dr Simon N. Waddington

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

    Article • Cell Reports, March 2016, Elsevier

    Dr Simon N. Waddington

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

    Article • Current Stem Cell Reports, February 2016, Springer Science + Business Media

    Dr Simon N. Waddington

  10. Gene Therapy with Adeno-associated Virus for Cystic Fibrosis

    Article • American Review of Respiratory Disease, February 2016, American Thoracic Society

    Dr Simon N. Waddington

  11. Proof-of-concept: neonatal intravenous injection of adeno-associated virus vectors results in successful transduction of myenteric and submucosal neurons in the mouse small and large intestine

    Article • Neurogastroenterology & Motility, November 2015, Wiley

    Professor Jean-Pierre Timmermans, Dr Simon N. Waddington

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

    Article • American Journal Of Pathology, September 2015, Elsevier

    Dr Simon N. Waddington

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

    Article • July 2015, Bioscientifica

    Dr Simon N. Waddington

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

    Article • Scientific Reports, July 2015, Nature

    Dr Simon N. Waddington

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

    Article • The FASEB Journal, June 2015, Federation of American Societies For Experimental Biology (FASEB)

    Dr Simon N. Waddington

  16. Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery

    Article • Journal of Immunology Research, January 2015, Hindawi Publishing Corporation

    Dr Simon N. Waddington

  17. Perinatal systemic gene delivery using adeno-associated viral vectors

    Article • Frontiers in Molecular Neuroscience, November 2014, Frontiers

    Dr Simon N. Waddington

  18. The Human Desmin Promoter Drives Robust Gene Expression for Skeletal Muscle Stem Cell-Mediated Gene Therapy

    Article • Current Gene Therapy, September 2014, Bentham Science Publishers

    Dr Simon N. Waddington

  19. Regionally-Specified Second Trimester Fetal Neural Stem Cells Reveals Differential Neurogenic Programming

    Article • PLoS ONE, September 2014, PLOS

    Dr Simon N. Waddington

  20. Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy

    Article • Molecular Therapy, July 2014, Nature

    Dr Simon N. Waddington

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

    Article • The FASEB Journal, February 2014, Federation of American Societies For Experimental Biology (FASEB)

    Dr Simon N. Waddington

  22. Pseudotyping the adenovirus serotype 5 capsid with both the fibre and penton of serotype 35 enhances vascular smooth muscle cell transduction

    Article • Gene Therapy, September 2013, Nature

    Dr Simon N. Waddington

  23. Transduction of Fetal Mice With a Feline Lentiviral Vector Induces Liver Tumors Which Exhibit an E2F Activation Signature

    Article • Molecular Therapy, August 2013, Nature

    Dr Simon N. Waddington

  24.  -Glucosidase 2 (GBA2) Activity and Imino Sugar Pharmacology

    Article • Journal of Biological Chemistry, July 2013, American Society for Biochemistry & Molecular Biology (ASBMB)

    Dr Simon N. Waddington

  25. Mitochondria and Quality Control Defects in a Mouse Model of Gaucher Disease—Links to Parkinson’s Disease

    Article • Cell Metabolism, June 2013, Elsevier

    Dr Simon N. Waddington

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

    Article • Journal of Inherited Metabolic Disease, February 2013, Springer Science + Business Media

    Dr Simon N. Waddington

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

    Article • Blood, February 2013, American Society of Hematology

    Dr Simon N. Waddington

  28. Exon Skipping of Hepatic APOB Pre-mRNA With Splice-switching Oligonucleotides Reduces LDL Cholesterol In Vivo

    Article • Molecular Therapy, January 2013, Nature

    Dr Simon N. Waddington

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

    Article • Journal of Inherited Metabolic Disease, November 2012, Springer Science + Business Media

    Dr Simon N. Waddington

  30. The Fetal Mouse Is a Sensitive Genotoxicity Model That Exposes Lentiviral-associated Mutagenesis Resulting in Liver Oncogenesis

    Article • Molecular Therapy, November 2012, Nature

    Dr Simon N. Waddington

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

    Article • Nature Immunology, October 2012, Springer Science + Business Media

    Susan M Lea, Professor Christian DROUET, Dr Simon N. Waddington

  32. The case for intrauterine gene therapy

    Article • Best Practice & Research Clinical Obstetrics & Gynaecology, October 2012, Elsevier

    Dr Simon N. Waddington

  33. Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems

    Article • Gene Therapy, January 2012, Nature

    Dr Simon N. Waddington

  34. Prenatal Gene Therapy

    Article • January 2012, Springer Science + Business Media

    Dr Simon N. Waddington

  35. Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Use of Large Animal Models with Relevance to Human Application

    Article • January 2012, Springer Science + Business Media

    Dr Simon N. Waddington

  36. Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Mouse Models for Developmental Aberrations and Inadvertent Germ Line Transmission

    Article • January 2012, Springer Science + Business Media

    Dr Simon N. Waddington

  37. Choice of Surrogate and Physiological Markers for Prenatal Gene Therapy

    Article • January 2012, Springer Science + Business Media

    Dr Simon N. Waddington

  38. Animal Models for Prenatal Gene Therapy: Rodent Models for Prenatal Gene Therapy

    Article • January 2012, Springer Science + Business Media

    Dr Simon N. Waddington

  39. Candidate Diseases for Prenatal Gene Therapy

    Article • January 2012, Springer Science + Business Media

    Dr Simon N. Waddington

  40. The Concept of Prenatal Gene Therapy

    Article • January 2012, Springer Science + Business Media

    Dr Simon N. Waddington

  41. Vector Systems for Prenatal Gene Therapy: Choosing Vectors for Different Applications

    Article • January 2012, Springer Science + Business Media

    Dr Simon N. Waddington

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

    Article • Blood, December 2011, American Society of Hematology

    Dr Simon N. Waddington

  43. In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression

    Article • Gene Therapy, November 2011, Nature

    Dr Simon N. Waddington

  44. Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques

    Article • Molecular Therapy, November 2011, Elsevier

    Dr Simon N. Waddington

  45. Perinatal Gene Transfer to the Liver

    Article • Current Pharmaceutical Design, August 2011, Bentham Science Publishers

    Dr Simon N. Waddington

  46. 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

    Article • The FASEB Journal, July 2011, Federation of American Societies For Experimental Biology (FASEB)

    Dr Simon N. Waddington

  47. Fetal gene therapy: recent advances and current challenges

    Article • Expert Opinion on Biological Therapy, May 2011, Informa Healthcare

    Dr Simon N. Waddington

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

    Article • Prenatal Diagnosis, May 2011, Wiley

    Dr Simon N. Waddington

  49. Recombinant Adeno-Associated Virus-Mediated In Utero Gene Transfer Gives Therapeutic Transgene Expression in the Sheep

    Article • Human Gene Therapy, April 2011, Mary Ann Liebert Inc

    Dr Simon N. Waddington

  50. Recent advances in fetal gene therapy

    Article • Therapeutic Delivery, April 2011, Future Science

    Dr Simon N. Waddington

  51. Perinatal gene delivery to the CNS

    Article • Therapeutic Delivery, April 2011, Future Science

    Dr Simon N. Waddington

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

    Article • Journal of Molecular Medicine, February 2011, Springer Science + Business Media

    Dr Simon N. Waddington

  53. 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

    Article • Molecular Therapy, January 2011, Nature

    Dr Simon N. Waddington

  54. In utero gene transfer to the mouse nervous system: Figure 1

    Article • Biochemical Society Transactions, December 2010, Portland Press Ltd.

    Dr Simon N. Waddington

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

    Article • Biochemical Society Transactions, December 2010, Portland Press Ltd.

    Dr Simon N. Waddington

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

    Article • Blood, November 2010, American Society of Hematology

    Dr Simon N. Waddington

  57. Gene Delivery of a Mutant TGFβ3 Reduces Markers of Scar Tissue Formation After Cutaneous Wounding

    Article • Molecular Therapy, August 2010, Nature

    Dr Simon N. Waddington

  58. 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

    Article • Blood, August 2010, American Society of Hematology

    Dr Simon N. Waddington

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

    Article • Blood, July 2010, American Society of Hematology

    Dr Simon N. Waddington

  60. Neonatal Gene Therapy of Glycogen Storage Disease Type Ia Using a Feline Immunodeficiency Virus–based Vector

    Article • Molecular Therapy, June 2010, Nature

    Dr Simon N. Waddington

  61. Increased Secretion of Lipoproteins in Transgenic Mice Expressing Human D374Y PCSK9 Under Physiological Genetic Control

    Article • Arteriosclerosis Thrombosis and Vascular Biology, May 2010, Wolters Kluwer Health

    Dr Simon N. Waddington

  62. Desmin-regulated Lentiviral Vectors for Skeletal Muscle Gene Transfer

    Article • Molecular Therapy, March 2010, Elsevier

    Dr Simon N. Waddington

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

    Article • The Analyst, January 2010, Royal Society of Chemistry

    Dr Simon N. Waddington

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

    Article • International Archives of Medicine, January 2010, Springer Science + Business Media

    Dr Simon N. Waddington

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

    Article • Glia, January 2010, Wiley

    Dr Simon N. Waddington

  66. Erratum to “Influence of Coagulation Factor X on In Vitro and In Vivo Gene Delivery by Adenovirus (Ad) 5, Ad35, and Chimeric Ad5/Ad35 Vectors”

    Article • Molecular Therapy, October 2009, Nature

    Dr Simon N. Waddington, Professor David Bhella

  67. Influence of Coagulation Factor X on In Vitro and In Vivo Gene Delivery by Adenovirus (Ad) 5, Ad35, and Chimeric Ad5/Ad35 Vectors

    Article • Molecular Therapy, July 2009, Nature

    Dr Simon N. Waddington, Professor David Bhella

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

    Article • Blood, May 2009, American Society of Hematology

    Dr Simon N. Waddington, Professor David Bhella

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

    Article • Cell Cycle, April 2009, Taylor & Francis

    Dr Simon N. Waddington

  70. The Cyclopentenone 15-Deoxy-Δ12,14-Prostaglandin J2Delays Lipopolysaccharide-Induced Preterm Delivery and Reduces Mortality in the Newborn Mouse

    Article • Endocrinology, February 2009, Endocrine Society

    Dr Simon N. Waddington

  71. Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors

    Article • Gene Therapy, January 2009, Nature

    Dr Simon N. Waddington

  72. Effect of Neutralizing Sera on Factor X-Mediated Adenovirus Serotype 5 Gene Transfer

    Article • Journal of Virology, October 2008, ASM Journals

    Dr Simon N. Waddington

  73. Luciferin Detection After Intranasal Vector Delivery Is Improved by Intranasal Rather Than Intraperitoneal Luciferin Administration

    Article • Human Gene Therapy, October 2008, Mary Ann Liebert Inc

    Dr Simon N. Waddington

  74. Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector

    Article • Gene Therapy, July 2008, Nature

    Dr Simon N. Waddington

  75. Genetic aspects and research development in haemostasis

    Article • Haemophilia, July 2008, Wiley

    Dr Simon N. Waddington

  76. Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression

    Article • Gene Therapy, April 2008, Nature

    Dr Simon N. Waddington

  77. Intra-amniotic Delivery of CFTR-expressing Adenovirus Does Not Reverse Cystic Fibrosis Phenotype in Inbred CFTR-knockout Mice

    Article • Molecular Therapy, March 2008, Nature

    Dr Simon N. Waddington

  78. Adenovirus Serotype 5 Hexon Mediates Liver Gene Transfer

    Article • Cell, February 2008, Elsevier

    Dr Simon N. Waddington, Professor David Bhella

  79. Stable Gene Transfer to Muscle Using Non-integrating Lentiviral Vectors

    Article • Molecular Therapy, August 2007, Nature

    Dr Simon N. Waddington

  80. The Influence of Blood on In Vivo Adenovirus Bio-distribution and Transduction

    Article • Molecular Therapy, August 2007, Elsevier

    Dr Simon N. Waddington

  81. 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

    Article • Journal of Virology, June 2007, ASM Journals

    Dr Simon N. Waddington

  82. Widespread Distribution and Muscle Differentiation of Human Fetal Mesenchymal Stem Cells After Intrauterine Transplantation in DystrophicmdxMouse

    Article • Stem Cells, April 2007, Wiley

    Dr Simon N. Waddington

  83. Influence of Coagulation Factor Zymogens on the Infectivity of Adenoviruses Pseudotyped with Fibers from Subgroup D

    Article • Journal of Virology, January 2007, ASM Journals

    Dr Simon N. Waddington

  84. Delivery and long-term expression of a 135 kbLDLR genomic DNA locusin vivo by hydrodynamic tail vein injection

    Article • The Journal of Gene Medicine, January 2007, Wiley

    Dr Simon N. Waddington

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

    Article • Blood, October 2006, American Society of Hematology

    Dr Simon N. Waddington

  86. Evaluation of prenatal intra-amniotic LAMB3 gene delivery in a mouse model of Herlitz disease

    Article • Gene Therapy, July 2006, Nature

    Dr Simon N. Waddington

  87. Clinically Applicable Procedure for Gene Delivery to Fetal Gut by Ultrasound-Guided Gastric Injection: Toward Prenatal Prevention of Early-Onset Intestinal Diseases

    Article • Human Gene Therapy, July 2006, Mary Ann Liebert Inc

    Dr Simon N. Waddington

  88. 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

    Article • Blood, April 2006, American Society of Hematology

    Dr Simon N. Waddington

  89. Corrigendum to "Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice"

    Article • Molecular Therapy, April 2006, Nature

    Dr Simon N. Waddington

  90. Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice

    Article • Molecular Therapy, October 2005, Nature

    Dr Simon N. Waddington

  91. Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX

    Article • Gene Therapy, September 2005, Nature

    Dr Simon N. Waddington

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

    Article • American Journal of Obstetrics and Gynecology, September 2005, Elsevier

    Dr Simon N. Waddington

  93. Gene Therapy Progress and Prospects: Fetal gene therapy – first proofs of concept – some adverse effects

    Article • Gene Therapy, September 2005, Nature

    Dr Simon N. Waddington

  94. Factors Influencing Adenovirus-Mediated Airway Transduction in Fetal Mice

    Article • Molecular Therapy, September 2005, Nature

    Dr Simon N. Waddington

  95. In Utero gene therapy: current challenges and perspectives

    Article • Molecular Therapy, May 2005, Elsevier

    Dr Simon N. Waddington

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

    Article • The Journal of Gene Medicine, January 2005, Wiley

    Dr Simon N. Waddington

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

    Article • The Journal of Gene Medicine, January 2005, Wiley

    Dr Simon N. Waddington

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

    Article • Blood, November 2004, American Society of Hematology

    Dr Simon N. Waddington

  99. Fetal and neonatal gene therapy: benefits and pitfalls

    Article • Gene Therapy, October 2004, Nature

    Dr Simon N. Waddington

  100. Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy

    Article • Gene Therapy, May 2004, Nature

    Dr Simon N. Waddington

  101. Reduced toxicity of F-deficient Sendai virus vector in the mouse fetus

    Article • Gene Therapy, January 2004, Nature

    Dr Simon N. Waddington

  102. Widespread and efficient marker gene expression in the airway epithelia of fetal sheep after minimally invasive tracheal application of recombinant adenovirus in utero

    Article • Gene Therapy, January 2004, Nature

    Dr Simon N. Waddington

  103. The Hopes and Fears of In Utero Gene Therapy for Genetic Disease—A Review

    Article • Placenta, October 2003, Elsevier

    Dr Simon N. Waddington

  104. Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice

    Article • Gene Therapy, August 2003, Nature

    Dr Simon N. Waddington

  105. Ultrasound-Guided Percutaneous Delivery of Adenoviral Vectors Encoding the -Galactosidase and Human Factor IX Genes to Early Gestation Fetal Sheep In Utero

    Article • Human Gene Therapy, March 2003, Mary Ann Liebert Inc

    Dr Simon N. Waddington

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

    Article • Blood, October 2002, American Society of Hematology

    Dr Simon N. Waddington

  107. Arginase in glomerulonephritis

    Article • Kidney International, March 2002, Elsevier

    Dr Simon N. Waddington

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

    Article • The Journal of Gene Medicine, January 2002, Wiley

    Dr Simon N. Waddington

  109. Arginase in Glomerulonephritis

    Article • Nephron Experimental Nephrology, May 2000, Karger Publishers

    Dr Simon N. Waddington

  110. Induced nitric oxide (NO) synthesis in heterologous nephrotoxic nephritis; effects of selective inhibition in neutrophil-dependent glomerulonephritis

    Article • Clinical & Experimental Immunology, November 1999, Wiley

    Dr Simon N. Waddington

  111. Arginase AI Is Upregulated in Acute Immune Complex-Induced Inflammation

    Article • Biochemical and Biophysical Research Communications, June 1998, Elsevier

    Dr Simon N. Waddington

  112. Anti-GBM glomerulonephritis in mice lacking nitric oxide synthase type 2 Rapid Communication

    Article • Kidney International, April 1998, Elsevier

    Dr Simon N. Waddington