Simon N. Waddington

South African Medical Research Council — South Africa

Biochemistry, Genetics and Molecular Biology · Life Sciences

54h-index11.2kcitations384works3.62yr avg
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Research Topics

Virus-based gene therapy research(154), CRISPR and Genetic Engineering(67), RNA Interference and Gene Delivery(42), Viral gastroenteritis research and epidemiology(39), Health, Medicine and Society(37)

Publications383 total

Modeling Ascending Vaginal Infection, Preterm Birth, and Neonatal Morbidity in Mice
Journal of Visualized Experiments·2025
Genetic therapies for neurological diseases
Pharmacological Reviews·2025Open Access
Next Generation AAV-F Capsid gene therapy rescues disease pathology in a model of Pyruvate Dehydrogenase Complex Deficiency
2025· 1 citedOpen Access
AAV delivery of RNA editing machinery rescues SUDEP and seizure phenotype in a mouse model of Dravet Syndrome
2025Open Access
Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy
Pharmaceutics·2025Open Access
Genetic therapies for movement disorders - current status
Journal of Neurology·2025· 4 citedOpen Access
Ascending Vaginal Infection in Mice Induces Preterm Birth and Neonatal Morbidity
American Journal Of Pathology·2025· 2 citedOpen Access
Contaminating manufacturing plasmids and disrupted vector genomes present in liver tissue following adeno-associated virus gene therapy
2025Open Access
Unifying biology of neurodegeneration in lysosomal storage diseases
Journal of Inherited Metabolic Disease·2025· 8 citedOpen Access
Gene therapy prevents hepatic mitochondrial dysfunction in murine deoxyguanosine kinase deficiency
Molecular Therapy — Methods & Clinical Development·2024· 3 citedOpen Access
Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice
Cells·2024· 2 citedOpen Access
AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet Syndrome
2024Open Access
Author response: Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
2024Open Access
Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
eLife·2024· 1 citedOpen Access
Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
2024Open Access
Reviewer #1 (Public Review): Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
2024Open Access
Author response: Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
2024Open Access
Reviewer #2 (Public Review): Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
2024Open Access
Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model
Scientific Reports·2024· 15 citedOpen Access
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease
Human Molecular Genetics·2024· 3 citedOpen Access
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Frequent Co-authors

Suzanne M. K. Buckley(16), John H. McVey(11), Andrew H. Baker(8), Rahimin Affandi Abdul Rahim(8), Michael Themis(8), Charles Coutelle(8), Donald Peebles(8), Alan L. Parker(7), Stuart A. Nicklin(7), Adrian J. Thrasher(7), Amit C. Nathwani(6), Jenny McIntosh(6), Megha Nivsarkar(5), Bronwen Herbert(5), Jerry Kok Yen Chan(5), Nico van Rooijen(4), Menzo Havenga(4), John T. Gray(4), Edward G. D. Tuddenham(4), Andrew M. Davidoff(4)