Simon N. Waddington

EGA Institute for Women's Health, University College London

Affiliated withUniversity College London

Research Area

Biography

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JoVE Journal Publications

ArticleTotal : 1	Year
Publication title	2025

Other Publications

Article	Year
Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy. American journal of obstetrics and gynecology\| PubMed ID: 16157120	2005
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 li... Blood\| PubMed ID: 16322469	2006
Fetal gene transfer. Current opinion in molecular therapeutics\| PubMed ID: 17932807	2007
Activation and deactivation of periventricular white matter phagocytes during postnatal mouse development. Glia\| PubMed ID: 19544386	2010
Desmin-regulated lentiviral vectors for skeletal muscle gene transfer. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 19935780	2010
The differentiation and engraftment potential of mouse hematopoietic stem cells is maintained after bio-electrospray. The Analyst\| PubMed ID: 20024196	2010
Gene delivery of a mutant TGFβ3 reduces markers of scar tissue formation after cutaneous wounding. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 20736928	2010
Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood\| PubMed ID: 21041718	2011
In utero gene transfer to the mouse nervous system. Biochemical Society transactions\| PubMed ID: 21118113	2010
LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives. International archives of medicine\| PubMed ID: 21144047	2010
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. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 21245849	2011
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 21629224	2011
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 journal : official publication of the Federation of American Societies for Experimental Biology\| PubMed ID: 21746868	2011
Perinatal gene transfer to the liver. Current pharmaceutical design\| PubMed ID: 21774770	2011
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage. Blood\| PubMed ID: 22134170	2012
Candidate diseases for prenatal gene therapy. Methods in molecular biology (Clifton, N.J.)\| PubMed ID: 22648766	2012
Choice of surrogate and physiological markers for prenatal gene therapy. Methods in molecular biology (Clifton, N.J.)\| PubMed ID: 22648777	2012
Monitoring for potential adverse effects of prenatal gene therapy: use of large animal models with relevance to human application. Methods in molecular biology (Clifton, N.J.)\| PubMed ID: 22648778	2012
Perinatal gene delivery to the CNS. Therapeutic delivery\| PubMed ID: 22826856	2011
The CD46-Jagged1 interaction is critical for human T(H)1 immunity. Nature immunology\| PubMed ID: 23086448	2012
The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 23299800	2013
Mitochondria and quality control defects in a mouse model of Gaucher disease--links to Parkinson's disease. Cell metabolism\| PubMed ID: 23707074	2013
β-Glucosidase 2 (GBA2) activity and imino sugar pharmacology. The Journal of biological chemistry\| PubMed ID: 23880767	2013
Transduction of fetal mice with a feline lentiviral vector induces liver tumors which exhibit an E2F activation signature. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 23982166	2014
Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 25034357	2014
Regionally-specified second trimester fetal neural stem cells reveals differential neurogenic programming. PloS one\| PubMed ID: 25181041	2014
Perinatal systemic gene delivery using adeno-associated viral vectors. Frontiers in molecular neuroscience\| PubMed ID: 25452713	2014
Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector delivery. Journal of immunology research\| PubMed ID: 25759840	2015
Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates. FASEB journal : official publication of the Federation of American Societies for Experimental Biology\| PubMed ID: 26062602	2015
In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters. Scientific reports\| PubMed ID: 26138224	2015
Specific inhibition of c-Jun N-terminal kinase delays preterm labour and reduces mortality. Reproduction (Cambridge, England)\| PubMed ID: 26183892	2015
Specific Lipopolysaccharide Serotypes Induce Differential Maternal and Neonatal Inflammatory Responses in a Murine Model of Preterm Labor. The American journal of pathology\| PubMed ID: 26212908	2015
Gene Therapy with Adeno-associated Virus for Cystic Fibrosis. American journal of respiratory and critical care medicine\| PubMed ID: 26829420	2016
NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming. Cell reports\| PubMed ID: 26904936	2016
Modeling hormonal and inflammatory contributions to preterm and term labor using uterine temporal transcriptomics. BMC medicine\| PubMed ID: 27291689	2016
A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells. Current stem cell reports\| PubMed ID: 27398291	2016
Regulation of post-Golgi LH3 trafficking is essential for collagen homeostasis. Nature communications\| PubMed ID: 27435297	2016
The Local and Systemic Immune Response to Intrauterine LPS in the Prepartum Mouse. Biology of reproduction\| PubMed ID: 27760748	2016
Vps33b is crucial for structural and functional hepatocyte polarity. Journal of hepatology\| PubMed ID: 28082148	2017
Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice. Scientific reports\| PubMed ID: 28157201	2017
Lentiviral vectors can be used for full-length dystrophin gene therapy. Scientific reports\| PubMed ID: 28250438	2017
Expanding the phenotype in argininosuccinic aciduria: need for new therapies. Journal of inherited metabolic disease\| PubMed ID: 28251416	2017
Lentiviral vectors can be used for full-length dystrophin gene therapy. Scientific reports\| PubMed ID: 28303972	2017
In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 28462816	2017
Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 28550974	2017
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. Journal of inherited metabolic disease\| PubMed ID: 28567541	2017
The power of bioluminescence imaging in understanding host-pathogen interactions. Methods (San Diego, Calif.)\| PubMed ID: 28694065	2017
Continual conscious bioluminescent imaging in freely moving somatotransgenic mice. Scientific reports\| PubMed ID: 28743959	2017
Bioluminescence Monitoring of Promoter Activity In Vitro and In Vivo. Methods in molecular biology (Clifton, N.J.)\| PubMed ID: 28801899	2017
Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors. Experimental hematology\| PubMed ID: 28911908	2018
Progesterone, the maternal immune system and the onset of parturition in the mouse. Biology of reproduction\| PubMed ID: 29145579	2018
High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors. Journal of controlled release : official journal of the Controlled Release Society\| PubMed ID: 29289570	2018
A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model. Experimental hematology\| PubMed ID: 29605545	2018
NF-κB Activity Initiates Human ESC-Derived Neural Progenitor Cell Differentiation by Inducing a Metabolic Maturation Program. Stem cell reports\| PubMed ID: 29681545	2018
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency. Brain : a journal of neurology\| PubMed ID: 29788236	2018
AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease. Human molecular genetics\| PubMed ID: 29878115	2018
Fetal gene therapy for neurodegenerative disease of infants. Nature medicine\| PubMed ID: 30013199	2018
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort. Human gene therapy\| PubMed ID: 30027761	2019
Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice. The American journal of pathology\| PubMed ID: 30036519	2018
Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice. Molecular therapy. Nucleic acids\| PubMed ID: 30081233	2018
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer. Nature communications\| PubMed ID: 30158522	2018
Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques. FASEB journal : official publication of the Federation of American Societies for Experimental Biology\| PubMed ID: 30517034	2019
Production of lentiviral vectors using novel, enzymatically produced, linear DNA. Gene therapy\| PubMed ID: 30643205	2019
Fetal gene therapy for neurodegenerative lysosomal storage diseases. Journal of inherited metabolic disease\| PubMed ID: 30715735	2019
In Utero Gene Therapy Consensus Statement from the IFeTIS. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 30837116	2019
In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques. Scientific reports\| PubMed ID: 31406195	2019
Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload. Cell death and differentiation\| PubMed ID: 31685979	2020
Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment. Metabolites\| PubMed ID: 31718089	2019
Continual Conscious Bioluminescent Imaging in Freely Moving Mice. Methods in molecular biology (Clifton, N.J.)\| PubMed ID: 31721124	2020
Impaired folate 1-carbon metabolism causes formate-preventable hydrocephalus in glycine decarboxylase-deficient mice. The Journal of clinical investigation\| PubMed ID: 31794432	2020
Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Te... Scientific reports\| PubMed ID: 31874968	2019
Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes. Human molecular genetics\| PubMed ID: 31919491	2020
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors. Scientific reports\| PubMed ID: 32034258	2020
Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth. Frontiers in immunology\| PubMed ID: 32117260	2020
Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload. Cell death and differentiation\| PubMed ID: 32152554	2020
Fetal and Maternal Safety Considerations for In Utero Therapy Clinical Trials: iFeTiS Consensus Statement. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 33069884	2020
Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation. Molecular therapy. Methods & clinical development\| PubMed ID: 33553484	2021
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development. Biomolecules\| PubMed ID: 33924076	2021
Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism. Science translational medicine\| PubMed ID: 34011628	2021
In Vitro and In Vivo Evaluation of Human Adenovirus Type 49 as a Vector for Therapeutic Applications. Viruses\| PubMed ID: 34452348	2021
Rapid and inexpensive purification of adenovirus vectors using an optimised aqueous two-phase technology. Journal of virological methods\| PubMed ID: 34626684	2022
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys. Molecular therapy. Methods & clinical development\| PubMed ID: 34703837	2021
HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival. Gene therapy\| PubMed ID: 35513551	2022
Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors. Nanoscale\| PubMed ID: 36866741	2023
Genomic investigations of unexplained acute hepatitis in children. Nature\| PubMed ID: 36996872	2023
Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases. Movement disorders : official journal of the Movement Disorder Society\| PubMed ID: 37147851	2023
Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches. Cells\| PubMed ID: 37443770	2023
Fetal gene therapy. Journal of inherited metabolic disease\| PubMed ID: 37470194	2024
Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease. EMBO molecular medicine\| PubMed ID: 37642150	2023
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria. Science translational medicine\| PubMed ID: 38198573	2024
Gene therapy for neurotransmitter-related disorders. Journal of inherited metabolic disease\| PubMed ID: 38221762	2024
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease. Human molecular genetics\| PubMed ID: 38757200	2024
Dysregulated Wnt and NFAT signaling in a Parkinson's disease LRRK2 G2019S knock-in model. Scientific reports\| PubMed ID: 38811759	2024
Human gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome. eLife\| PubMed ID: 38990208	2024
Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice. Cells\| PubMed ID: 39682727	2024
Unifying biology of neurodegeneration in lysosomal storage diseases. Journal of inherited metabolic disease\| PubMed ID: 39822020	2025
Ascending Vaginal Infection in Mice Induces Preterm Birth and Neonatal Morbidity. The American journal of pathology\| PubMed ID: 39892780	2025
Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy. Pharmaceutics\| PubMed ID: 40430940	2025