Fetal gene therapy for neurodegenerative disease of infants von Massaro, Giulia, Mattar, Citra N. Z., Wong, Andrew M. S., Sirka, Ernestas, Buckley, Suzanne M. K., Herbert, Bronwen R., Karlsson, Stefan, Perocheau, Dany P., Burke, Derek, Heales, Simon, Richard-Londt, Angela, Brandner, Sebastian, Huebecker, Mylene, Priestman, David A., Platt, Frances M., Mills, Kevin, Biswas, Arijit, Cooper, Jonathan D., Chan, Jerry K. Y., Cheng, Seng H., Waddington, Simon N., Rahim, Ahad A.

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Codon optimization of human factor VIII cDNAs leads to high-level expression von Ward, Natalie J., Buckley, Suzanne M.K., Waddington, Simon N., VandenDriessche, Thierry, Chuah, Marinee K.L., Nathwani, Amit C., McIntosh, Jenny, Tuddenham, Edward G.D., Kinnon, Christine, Thrasher, Adrian J., McVey, John H.

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Biodistribution and retargeting of FX-binding ablated adenovirus serotype 5 vectors von Alba, Raul, Bradshaw, Angela C., Coughlan, Lynda, Denby, Laura, McDonald, Robert A., Waddington, Simon N., Buckley, Suzanne M.K., Greig, Jenny A., Parker, Alan L., Miller, Ashley M., Wang, Hongjie, Lieber, Andre, van Rooijen, Nico, McVey, John H., Nicklin, Stuart A., Baker, Andrew H.

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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 von Rahim, Ahad A., Wong, Andrew M. S., Hoefer, Klemens, Buckley, Suzanne M. K., Mattar, Citra N., Cheng, Seng H., Chan, Jerry K. Y., Cooper, Jonathan D., Waddington, Simon N.

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Effect of Neutralizing Sera on Factor X-Mediated Adenovirus Serotype 5 Gene Transfer von PARKER, Alan L, WADDINGTON, Simon N, BUCKLEY, Suzanne M. K, CUSTERS, Jerome, HAVENGA, Menzo J. E, VAN ROOIJEN, Nico, GOUDSMIT, Jaap, MCVEY, John H, NICKLIN, Stuart A, BAKER, Andrew H

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Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors von 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, Waddington, S N, Thrasher, A J, Raivich, G

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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 von WADDINGTON, Simon N, PARKER, Alan L, HAVENGA, Menzo, NICKLIN, Stuart A, BUCKLEY, Suzanne M. K, MCVEY, John H, BAKER, Andrew H

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Local delivery of VEGF adenovirus to the uterine artery increases vasorelaxation and uterine blood flow in the pregnant sheep von David, A L, Torondel, B, Zachary, I, Wigley, V, Nader, K A, Mehta, V, Buckley, S M K, Cook, T, Boyd, M, Rodeck, C H, Martin, J, Peebles, D M

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Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer von Baruteau, Julien, Perocheau, Dany P., Hanley, Joanna, Lorvellec, Maëlle, Rocha-Ferreira, Eridan, Karda, Rajvinder, Ng, Joanne, Suff, Natalie, Diaz, Juan Antinao, Rahim, Ahad A., Hughes, Michael P., Banushi, Blerida, Prunty, Helen, Hristova, Mariya, Ridout, Deborah A., Virasami, Alex, Heales, Simon, Howe, Stewen J., Buckley, Suzanne M. K., Mills, Philippa B., Gissen, Paul, Waddington, Simon N.

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Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth von Suff, Natalie, Karda, Rajvinder, Diaz, Juan Antinao, Ng, Joanne, Baruteau, Julien, Perocheau, Dany, Taylor, Peter W, Alber, Dagmar, Buckley, Suzanne M K, Bajaj-Elliott, Mona, Waddington, Simon N, Peebles, Donald

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Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery von Waddington, Simon N., Rahim, Ahad A., McKay, Tristan R., Perocheau, Dany, Parker, Alan L., Buckley, Suzanne M. K., Nivsarkar, Megha S., Howe, Steven J.

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Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression von Buckley, S M K, Howe, S J, Sheard, V, Ward, N J, Coutelle, C, Thrasher, A J, Waddington, S N, McKay, T R

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Continual conscious bioluminescent imaging in freely moving somatotransgenic mice von Karda, Rajvinder, Perocheau, Dany P., Suff, Natalie, Ng, Joanne, Delhove, Juliette M. K. M., Buckley, Suzanne M. K., Richards, Samantha, Counsell, John R., Hagberg, Henrik, Johnson, Mark R., McKay, Tristan R., Waddington, Simon N.

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Perinatal systemic gene delivery using adeno-associated viral vectors von Karda, Rajvinder, Buckley, Suzanne M K, Mattar, Citra N, Ng, Joanne, Massaro, Giulia, Hughes, Michael P, Kurian, Manju A, Baruteau, Julien, Gissen, Paul, Chan, Jerry K Y, Bacchelli, Chiara, Waddington, Simon N, Rahim, Ahad A

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Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy von Vink, Conrad A., Counsell, John R., Perocheau, Dany P., Karda, Rajvinder, Buckley, Suzanne M.K., Brugman, Martijn H., Galla, Melanie, Schambach, Axel, McKay, Tristan R., Waddington, Simon N., Howe, Steven J.

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Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors von Karda, Rajvinder, Rahim, Ahad A., Wong, Andrew M. S., Suff, Natalie, Diaz, Juan Antinao, Perocheau, Dany P., Tijani, Maha, Ng, Joanne, Baruteau, Julien, Martin, Nuria Palomar, Hughes, Michael, Delhove, Juliette M. K. M., Counsell, John R., Cooper, Jonathan D., Henckaerts, Els, Mckay, Tristan R., Buckley, Suzanne M. K., Waddington, Simon N.

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In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor von Waddington, Simon N., Buckley, Suzanne M.K., Nivsarkar, Megha, Jezzard, Sarah, Schneider, Holm, Dahse, Thomas, Kemball-Cook, Geoff, Miah, Maznu, Tucker, Nick, Dallman, Margaret J., Themis, Mike, Coutelle, Charles

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Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late‐gestation nonhuman primates von Mattar, Citra N., Wong, Andrew M. S., Hoefer, Klemens, Alonso‐Ferrero, Maria E., Buckley, Suzanne M. K., Howe, Steven J., Cooper, Jonathan D., Waddington, Simon N., Chan, Jerry K. Y., Rahim, Ahad A.

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In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression von Rahim, A A, Wong, A M, Ahmadi, S, Hoefer, K, Buckley, S M K, Hughes, D A, Nathwani, A N, Baker, A H, McVey, J H, Cooper, J D, Waddington, S N

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AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage von Binny, Christopher, McIntosh, Jenny, Della Peruta, Marco, Kymalainen, Hanna, Tuddenham, Edward G.D., Buckley, Suzanne M.K., Waddington, Simon N., McVey, John H., Spence, Yunyu, Morton, Christopher L., Thrasher, Adrian J., Gray, John T., Castellino, Francis J., Tarantal, Alice F., Davidoff, Andrew M., Nathwani, Amit C.

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