256. A Novel Rationally Designed AAV Capsid Yields a Potent Neurotropic Gene Therapy Vector
Henckaerts, Els, Tordo, Julie, Palomar, Nuria, Rahman, Jahedur, Aldrin-Kirk, Patrick, Basche, Mark, Bennett, Antonette, O'Leary, Claire, Ali, Robin, Agbandje-McKenna, Mavis, Bigger, Brian, Rahim, Ahad A., Björklund, Tomas, Waddington, Simon, Linden, R. Michael
Published in Molecular therapy (01.05.2016)
Published in Molecular therapy (01.05.2016)
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305. Generation of Light-Producing, Somatic-Transgenic Mice Using Lentivirus and Adeno-Associated Virus Vectors
Karda, Rajvinder, Perocheau, Dany P., Buckley, Suzanne M.K., Delhove, Juliette M. K.M., Hughes, Michael, Rahim, Ahad A., Johnson, Mark R., Ng, Joanne, Sufi, Natalie, Mckay, Tristan R., Waddington, Simon N.
Published in Molecular therapy (01.05.2016)
Published in Molecular therapy (01.05.2016)
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Accumulation of damaged mitochondria in neuropathic Gaucher disease, the most prevalent risk factor for Parkinson's disease, is due to defective cellular degradation machinery
Osellame, Laura D., Rahim, Ahad, Gegg, Matthew E., Waddington, Simon, Schapira, Anthony H.V., Duchen, Michael R.
Published in Biochimica et biophysica acta. Bioenergetics (01.10.2012)
Published in Biochimica et biophysica acta. Bioenergetics (01.10.2012)
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Correction of Hemoglobin Levels in a Heterozygous Humanized Mouse Model of Thalassemia after Fetal Gene Therapy
Shangaris, Panicos, Loukogeorgakis, Stavros P, Subramaniam, Sindhu, Blundell, Mike, Bakhamis, Nahla, Liu, Shanrun, Eaton, Simon, Ramachandra, Durrgah, Maghsoudlou, Panayiotis, Urbani, Luca, Waddington, Simon, Archer, Joy, Antoniou, Mike, Thrasher, Adrian J, Ryan, Thomas, De Coppi, Paolo, David, Anna
Published in Blood (06.12.2014)
Published in Blood (06.12.2014)
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529. Novel LTR-1 Lentiviral Vectors Are Fully Functional Following the Removal of HIV-1 Gag-RRE Sequences
Counsell, John R., Vink, Conrad A., Perocheau, Dany P.B., Karda, Rajvinder, Ng, Joanne, Buckley, Suzanne M.K., Hubank, Michael, McKay, Tristan R., Delhove, Juliette M.K.M., Brugman, Martijn H., Waddington, Simon N., Howe, Steven J.
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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134. Generation of Light-Emitting Somatic-Transgenic Mice for Disease Modelling of Hypoxic Ischaemic Encephalopathy
Karda, Rajvinder, Delhove, Juliette M.K.M., Buckley, Suzanne M.K., Rahim, Ahad A., Rocha-Ferreira, Eridan, Perocheau, Dany P.B., Ng, Joanne, Mckay, Tristan R., Hagberg, Henrik H., Waddington, Simon N., Johnson, Mark R.
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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Organ targeted prenatal gene therapy—how far are we?: Fetal Therapy
MEHTA, Vedanta, KHALIL ABI NADER, WADDINGTON, Simon, DAVID, Anna L
Published in Prenatal diagnosis (2011)
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Published in Prenatal diagnosis (2011)
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Oncogenesis following vector delivery in the fetal mouse appears to be independent of WPRE-presence in vector constructs and supports this animal model for assessment of vector-associated genotoxicity
Nowrouzi, Ali, Cheung, Wing, Li, Tingting, Zhang, Xuegong, Arens, Anne, Paruzynski, Anna, Waddington, Simon N, Osejindu, Emma, Reja, Safia, von Kalle, Christof
Published in Molecular therapy (01.09.2012)
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Published in Molecular therapy (01.09.2012)
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Fetal gene transfer
Waddington, Simon N, Buckley, Suzanne Mk, David, Anna L, Peebles, Donald M, Rodeck, Charles H, Coutelle, Charles
Published in Current opinion in molecular therapeutics (01.10.2007)
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Published in Current opinion in molecular therapeutics (01.10.2007)
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Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant
McIntosh, Jenny, Lenting, Peter J, Tuddenham, Edward, Sotannde, Motunrayo, Waddington, Simon, Gray, John T., Davidoff, Andrew, Nathwani, Amit
Published in Blood (19.11.2010)
Published in Blood (19.11.2010)
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Designing for Inconsistency – The Dependency-Based PERICLES Approach
Vion-Dury, Jean-Yves, Lagos, Nikolaos, Kontopoulos, Efstratios, Riga, Marina, Mitzias, Panagiotis, Meditskos, Georgios, Waddington, Simon, Laurenson, Pip, Kompatsiaris, Ioannis
Published in New Trends in Databases and Information Systems (2015)
Published in New Trends in Databases and Information Systems (2015)
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The CD46-Jagged1 interaction is critical for human [T.sub.H]1 immunity
Le Friec, Gaelle, Sheppard, Devon, Whiteman, Pat, Karsten, Christian M, Shamoun, Salley Al-Tilib, Laing, Adam, Bugeon, Laurence, Dallman, Margaret J, Melchionna, Teresa, Chillakuri, Chandramouli, Smith, Richard A, Drouet, Christian, Couzi, Lionel, Fremeaux-Bacchi, Veronique, Kohl, Jorg, Waddington, Simon N, McDonnell, James M, Baker, Alastair, Handford, Penny A, Lea, Susan M, Kemper, Claudia
Published in Nature immunology (01.12.2012)
Published in Nature immunology (01.12.2012)
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The CD46-Jagged1 interaction is critical for human T sub(H)1 immunity
Le Friec, Gaeelle, Sheppard, Devon, Whiteman, Pat, Karsten, Christian M, Shamoun, Salley Al-Tilib, Laing, Adam, Bugeon, Laurence, Dallman, Margaret J, Melchionna, Teresa, Chillakuri, Chandramouli, Smith, Richard A, Drouet, Christian, Couzi, Lionel, Fremeaux-Bacchi, Veronique, Kohl, Jorg, Waddington, Simon N, McDonnell, James M, Baker, Alastair, Handford, Penny A, Lea, Susan M, Kemper, Claudia
Published in Nature immunology (01.12.2012)
Published in Nature immunology (01.12.2012)
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Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System
Binny, Christopher J, McIntosh, Jenny H, Tuddenham, Edward, Waddington, Simon, Buckley, Suzanne, Thrasher, Adrian, Gray, John T., Nathwani, Amit, Tarantal, Alice F, Davidoff, Andrew
Published in Blood (19.11.2010)
Published in Blood (19.11.2010)
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