Simon N Waddington

25PUBLICATIONS
105CO-AUTHORS
Gene and molecular therapyMolecular targetsNeurology and neuromuscular diseasesMedical biochemistry - amino acids and metabolitesPharmaceutical delivery technologies
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Publications (25)

|Jun 19, 2026
Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models.

|Jan 16, 2026
Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy.

Sarah Buddle, Li-An K Brown, Sofia Morfopoulou

|Jan 17, 2025
Unifying biology of neurodegeneration in lysosomal storage diseases.

Anna M Ludlaim, Simon N Waddington, Tristan R McKay

|Dec 17, 2024
Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice.

Loukia Touramanidou, Sonam Gurung, Claudiu A Cozmescu

|Jan 15, 2024
Gene therapy for neurotransmitter-related disorders.

Wing Sum Chu, Joanne Ng, Simon N Waddington

|Jan 10, 2024
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria.

Sonam Gurung, Oskar Vilhelmsson Timmermand, Dany Perocheau

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Frequent Collaborators

7 joint publications

John R Counsell

7 joint publications

Paul Gissen

6 joint publications

Julien Baruteau

5 joint publications

Rajvinder Karda