List of works by Simon N. Waddington

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

scientific article

A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.

scientific article published in July 2018

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

scientific article published on December 2011

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease

article

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

scientific article published in January 2010

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

scientific article published on 4 February 2014

Adenovirus serotype 5 hexon mediates liver gene transfer

scientific article published in February 2008

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

scientific article published on 19 October 2018

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

scientific article

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

scientific article published in April 1998

Arginase AI is upregulated in acute immune complex-induced inflammation

scientific article published on June 1998

Arginase in glomerulonephritis

scientific article published on 01 May 2000

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

scientific article published in Nature Communications

Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice

scientific article published on 21 July 2018

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

scientific article

Candidate diseases for prenatal gene therapy

scientific article published on January 2012

Choice of surrogate and physiological markers for prenatal gene therapy

scientific article

Clinically applicable procedure for gene delivery to fetal gut by ultrasound-guided gastric injection: toward prenatal prevention of early-onset intestinal diseases.

scientific article published in July 2006

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

scientific article published in November 2010

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

scientific article published in March 2005

Continual conscious bioluminescent imaging in freely moving somatotransgenic mice.

scientific article

Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload

scientific article published on 09 March 2020

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

scientific article published on 3 November 2005

Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis

scientific article published on December 2010

Delivering efficient liver-directed AAV-mediated gene therapy

scientific article published on 12 January 2017

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

scientific article published in June 2007

Desmin-regulated lentiviral vectors for skeletal muscle gene transfer

scientific article published on 24 November 2009

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

scientific article published on 8 February 2011

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

scientific article published on 9 April 2009

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

scientific article published on 22 October 2008

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

scientific article

Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy

scientific article published on 17 May 2017

Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors.

scientific article published on 11 September 2017

Erratum: Lentiviral vectors can be used for full-length dystrophin gene therapy

scientific article published on 29 August 2017

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

scientific article published on 27 July 2006

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

scientific article published on 31 January 2015

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

scientific article

Expanding the phenotype in argininosuccinic aciduria: need for new therapies

scientific article

Factors influencing adenovirus-mediated airway transduction in fetal mice.

scientific article

Fetal gene therapy for neurodegenerative disease of infants

article

Fetal gene therapy: recent advances and current challenges

scientific article published on 31 May 2011

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice

scholarly article by John R Counsell et al published 7 September 2018 in Molecular therapy. Nucleic acids

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.

scientific article

Gene Therapy with Adeno-associated Virus for Cystic Fibrosis

scientific article

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

scientific article

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.

scientific article

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors

scientific article published on 07 February 2020

Genetic aspects and research development in haemostasis.

scientific article published on July 2008

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

scientific article published on 2 November 2016

High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors

scientific article published on 28 December 2017

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

scientific article published on July 2004

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

scientific article

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

scientific article published on 08 May 2009

Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload

scientific article published on 04 November 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 article published on 12 August 2019

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.

scientific article published on 24 April 2017

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

scientific article

In utero gene therapy: current challenges and perspectives.

scientific article

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

scientific article published on 3 October 2002

In utero gene transfer to the mouse nervous system.

scientific article published on December 2010

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

scientific article published on 3 July 2015

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

scientific article

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

scientific article published on 6 May 2010

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

scientific article published on November 1999

Inducible nitric oxide synthase induction in Thy 1 glomerulonephritis is complement and reactive oxygen species dependent

scientific article published on 01 January 1999

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

scientific article

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

scientific article published on 24 January 2007

Intra-amniotic delivery of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice.

scientific article

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.

scientific article published on 11 July 2011

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

scientific article

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

scientific article published on 24 April 2008

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

scientific article

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

scientific article published on 17 March 2017

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.

scientific article

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

scientific article published in August 2003

Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice

scientific article published on 03 February 2017

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

scientific article

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

scientific journal article

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

scientific article

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

scientific article

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

scientific article

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

scientific article published on 20 June 2006

NF-κB Activity Initiates Human ESC-Derived Neural Progenitor Cell Differentiation by Inducing a Metabolic Maturation Program.

scientific article published on 17 April 2018

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

scientific article published on 16 February 2016

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

scientific article published on 22 June 2010

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

scientific article

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

scientific article

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

scientific article published on 25 May 2011

Perinatal systemic gene delivery using adeno-associated viral vectors

scientific article

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

scientific article

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

scientific article published in July 2004

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

scientific article

Progesterone, the maternal immune system and the onset of parturition in the mouse.

scientific article published on 14 November 2017

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

scientific article published on 12 November 2015

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

scientific article published on 5 September 2013

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 Techniques

scientific article published on 24 December 2019

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

scientific article published on 2 February 2011

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

scientific article published in April 2004

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

scientific article

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

scientific article (publication date: 20 July 2016)

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

scientific article

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

scientific article published on 16 July 2015

Stable gene transfer to muscle using non-integrating lentiviral vectors

scientific article published on 14 August 2007

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

scientific article published on 31 May 2011

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

scientific article

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

scientific article published on 26 January 2012

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

scientific article

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.

scientific article

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

scientific article

The CD46-Jagged1 interaction is critical for human TH1 immunity

scientific article

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

scientific article

The case for intrauterine gene therapy

scientific article published on 20 July 2012

The concept of prenatal gene therapy

scientific article published in January 2012

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

scientific article

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

scientific article published on 13 November 2009

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

scientific article published on 13 November 2012

The hopes and fears of in utero gene therapy for genetic disease--a review.

scientific article published on October 2003

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

scientific article published on January 2014

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

scientific article published on 15 May 2007

The power of bioluminescence imaging in understanding host-pathogen interactions.

scientific article

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

scientific article published on 20 February 2013

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

scientific article published on 28 August 2013

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

scientific article published in March 2003

Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment

scientific article published on 12 November 2019

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

scientific article published on January 2012

Vps33b is crucial for structural and functional hepatocyte polarity

scientific article published on 9 January 2017

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

scientific article published in January 2004

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

scientific article published on 21 December 2006

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

scientific article published on 23 July 2013

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