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Biology and Biochemistry

D-Index
59
Citations
11225
World Ranking
12667
National Ranking
984

Overview

Simon N. Waddington is a researcher affiliated with University College London in the United Kingdom. Their research spans key areas within biochemistry, genetics, and molecular biology, with a particular focus on molecular biology and genetics. The scientist has contributed extensively to the fields of clinical biochemistry, infectious diseases, and epidemiology.

Their main research topics include:

  • Virus-based gene therapy research
  • Metabolism and genetic disorders
  • Viral gastroenteritis research and epidemiology
  • Neonatal respiratory health research
  • Pancreatic function and diabetes
  • CRISPR and genetic engineering
  • Lysosomal storage disorders research

Recent papers authored or co-authored by Simon N. Waddington include:

  • Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development (2021, Biomolecules)
  • Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism (2021, Science Translational Medicine)
  • Fetal and Maternal Safety Considerations for In Utero Therapy Clinical Trials: iFeTiS Consensus Statement (2020, Molecular Therapy)
  • Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys (2021, Molecular Therapy - Methods & Clinical Development)
  • mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria (2024, Science Translational Medicine)

Simon N. Waddington frequently collaborates with other researchers including Rajvinder Karda, Dany Perocheau, Julien Baruteau, John R. Counsell, and Juan Antinao Díaz.

Their work has been commonly published in venues such as bioRxiv (Cold Spring Harbor Laboratory), Molecular Therapy - Methods & Clinical Development, Journal of Inherited Metabolic Disease, Science Translational Medicine, and EMBO Molecular Medicine.

The distribution of their publications highlights a significant engagement in preprint servers and specialized journals related to molecular and clinical therapies.

Best Publications

  • Adenovirus serotype 5 hexon mediates liver gene transfer

    Simon N. Waddington;John H. McVey;David Bhella;Alan L. Parker

  • 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

    Amit C. Nathwani;John T. Gray;Catherine Y. C. Ng;Junfang Zhou

  • 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.

    Amit C Nathwani;Amit C Nathwani;Cecilia Rosales;Jenny McIntosh;Ghasem Rastegarlari

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

    Laura D. Osellame;Ahad A. Rahim;Iain P. Hargreaves;Matthew E. Gegg;Matthew E. Gegg

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

    Alan L. Parker;Simon N. Waddington;Campbell G. Nicol;Dmitry M. Shayakhmetov

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

    Jenny McIntosh;Jenny McIntosh;Peter J. Lenting;Cecilia Rosales;Cecilia Rosales;Doyoung Lee

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

    Mike Themis;Simon N. Waddington;Manfred Schmidt;Christof von Kalle;Christof von Kalle

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

    Luis Apolonia;Simon N Waddington;Carolina Fernandes;Natalie J Ward

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

    Natalie J. Ward;Suzanne M. K. Buckley;Simon N. Waddington;Thierry VandenDriessche;Thierry VandenDriessche

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

    Raul Alba;Angela C. Bradshaw;Alan L. Parker;David Bhella

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

    Kate E. Hawkins;Shona Joy;Shona Joy;Juliette M.K.M. Delhove;Juliette M.K.M. Delhove;Juliette M.K.M. Delhove;Vassilios N. Kotiadis

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

    Gaëlle Le Friec;Devon Sheppard;Pat Whiteman;Christian M. Karsten

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

    Simon N Waddington;Megha S Nivsarkar;Ajay R Mistry;Suzanne M K Buckley

  • Fetal gene therapy for neurodegenerative disease of infants

    Giulia Massaro;Citra N. Z. Mattar;Andrew M. S. Wong;Ernestas Sirka

  • Widespread Distribution and Muscle Differentiation of Human Fetal Mesenchymal Stem Cells After Intrauterine Transplantation in Dystrophic mdx Mouse

    Jerry Chan;Simon N. Waddington;Keelin O'Donoghue;Hitoshi Kurata

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

    Simon N Waddington;Suzanne M K Buckley;Suzanne M K Buckley;Megha Nivsarkar;Megha Nivsarkar;Sarah Jezzard;Sarah Jezzard

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

    O Argyros;S P Wong;M Niceta;S N Waddington

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

    A. A. Rahim;A. M. S. Wong;S. J. Howe;S. M. K. Buckley

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

    Mariya Hristova;Daniel Cuthill;Virginia Zbarsky;Alejandro Acosta-Saltos

  • 36. Permanent Phenotypic Correction of Haemophilia B in Immunocompetent Mice by Prenatal Gene Therapy

    Simon N. Waddington;Megha S. Nivsarkar;Ajay R. Mistry;Suzanne M. K. Buckley

Frequent Co-Authors

Charles Coutelle
Charles Coutelle Imperial College London
Adrian J. Thrasher
Adrian J. Thrasher University College London
John H. McVey
John H. McVey University of Surrey
Amit C. Nathwani
Amit C. Nathwani University College London
Paul Gissen
Paul Gissen University College London
Jonathan D. Cooper
Jonathan D. Cooper Washington University in St. Louis
Stuart A. Nicklin
Stuart A. Nicklin University of Glasgow
Jerry Kok Yen Chan
Jerry Kok Yen Chan National University of Singapore
Ioannis Kompatsiaris
Ioannis Kompatsiaris Information Technologies Institute, Greece
Manju A. Kurian
Manju A. Kurian University College London

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