Waddington, Simon N

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

Nathwani, A. C., Rosales, C., McIntosh, J., Rastegarlari, G., Nathwani, D., Raj, D., Nawathe, S., Waddington, S. N., Bronson, R., Jackson, S., et al. (2011) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 345

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

McIntosh, J., Lenting, P. J., Rosales, C., Lee, D., Rabbanian, S., Raj, D., Patel, N., Tuddenham, E. G. D., Christophe, O. D., McVey, J. H., et al. (2013) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 278

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

Themis, M., Waddington, S. N., Schmidt, M., von Kalle, C., Wang, Y., Al-Allaf, F., Gregory, L. G., Nivsarkar, M., Themis, M., Holder, M. V., et al. (2005) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 243

Stable Gene Transfer to Muscle Using Non-integrating Lentiviral Vectors

Apolonia, L., Waddington, S. N., Fernandes, C., Ward, N. J., Bouma, G., Blundell, M. P., Thrasher, A. J., Collins, M. K., & Philpott, N. J. (2007) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 174

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

Tordo, J., O’Leary, C., Antunes, A. S. L. M., Palomar, N., Aldrin-Kirk, P., Basche, M., Bennett, A., D’Souza, Z., Gleitz, H., Godwin, A., et al. (2018) · Brain

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 113

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

Rahim, A. A., Wong, A. M. S., Howe, S. J., Buckley, S. M. K., Acosta-Saltos, A. D., Elston, K. E., Ward, N. J., Philpott, N. J., Cooper, J. D., Anderson, P. N., et al. (2009) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 101

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

Argyros, O., Wong, S. P., Niceta, M., Waddington, S. N., Howe, S. J., Coutelle, C., Miller, A. D., & Harbottle, R. P. (2008) · Gene Therapy

RNA Interference and Gene DeliveryVirus-based gene therapy research

Cited by 96

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

Perocheau, D. P., Cunningham, S. C., Lee, J., Antinao Diaz, J., Waddington, S. N., Gilmour, K., Eaglestone, S., Lisowski, L., Thrasher, A. J., Alexander, I. E., et al. (2019) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 81

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

Mattar, C. N., Wong, A. M. S., Hoefer, K., Alonso-Ferrero, M. E., Buckley, S. M. K., Howe, S. J., Cooper, J. D., Waddington, S. N., Chan, J. K. Y., & Rahim, A. A. (2015) · The FASEB Journal

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 46

Modifying inter-cistronic sequence significantly enhances IRES dependent second gene expression in bicistronic vector: Construction of optimised cassette for gene therapy of familial hypercholesterolemia

Al-Allaf, F. A., Abduljaleel, Z., Athar, M., Taher, M. M., Khan, W., Mehmet, H., Colakogullari, M., Apostolidou, S., Bigger, B., Waddington, S., et al. (2019) · Non-coding RNA Research

RNA and protein synthesis mechanismsRNA Interference and Gene Delivery

Cited by 18