Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches by Joanne Ng, Serena Barral, Simon N. Waddington, Manju A. Kurian

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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 by Natalie Suff, Natalie Suff, Natalie Suff, Rajvinder Karda, Juan Antinao Diaz, Joanne Ng, Julien Baruteau, Julien Baruteau, Dany Perocheau, Peter W. Taylor, Dagmar Alber, Suzanne M. K. Buckley, Mona Bajaj-Elliott, Mona Bajaj-Elliott, Simon N. Waddington, Simon N. Waddington, Donald Peebles

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Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development by Giulia Massaro, Amy F. Geard, Wenfei Liu, Oliver Coombe-Tennant, Simon N. Waddington, Julien Baruteau, Paul Gissen, Ahad A. Rahim

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Perinatal systemic gene delivery using adeno-associated viral vectors by Rajvinder eKarda, Suzanne M K Buckley, Citra N Mattar, Joanne eNg, Giulia eMassaro, Michael eHughes, Manju A Kurian, Julien eBaruteau, Paul eGissen, Jerry K Y Chan, Chiara eBacchelli, Simon N Waddington, Simon N Waddington, Ahad A Rahim

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Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment by Julien Baruteau, Youssef Khalil, Stephanie Grunewald, Marta Zancolli, Anupam Chakrapani, Maureen Cleary, James Davison, Emma Footitt, Simon N. Waddington, Paul Gissen, Philippa Mills

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Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation by John R. Counsell, Guillaume De Brabandere, Rajvinder Karda, Marc Moore, Antonio Greco, Alysha Bray, Juan Antinao Diaz, Dany P. Perocheau, Ulrike Mock, Simon N. Waddington

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Regionally-specified second trimester fetal neural stem cells reveals differential neurogenic programming. by Yiping Fan, Guillaume Marcy, Eddy S M Lee, Steve Rozen, Citra N Z Mattar, Simon N Waddington, Eyleen L K Goh, Mahesh Choolani, Jerry K Y Chan

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Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys by Julien Baruteau, Sharon C. Cunningham, Berna Seker Yilmaz, Dany P. Perocheau, Simon Eaglestone, Derek Burke, Adrian J. Thrasher, Simon N. Waddington, Leszek Lisowski, Ian E. Alexander, Paul Gissen

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Lentiviral vectors can be used for full-length dystrophin gene therapy by John R. Counsell, Zeinab Asgarian, Jinhong Meng, Veronica Ferrer, Conrad A. Vink, Steven J. Howe, Simon N. Waddington, Adrian J. Thrasher, Francesco Muntoni, Jennifer E. Morgan, Olivier Danos

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Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease by Valda Pauzuolyte, Aara Patel, James R Wawrzynski, Neil J Ingham, Yeh Chwan Leong, Rajvinder Karda, Maria Bitner‐Glindzicz, Wolfgang Berger, Simon N Waddington, Karen P Steel, Jane C Sowden

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In Vitro and In Vivo Evaluation of Human Adenovirus Type 49 as a Vector for Therapeutic Applications by Emily A. Bates, John R. Counsell, Sophie Alizert, Alexander T. Baker, Natalie Suff, Ashley Boyle, Angela C. Bradshaw, Simon N. Waddington, Stuart A. Nicklin, Andrew H. Baker, Alan L. Parker

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

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NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming by Kate E. Hawkins, Shona Joy, Juliette M.K.M. Delhove, Vassilios N. Kotiadis, Emilio Fernandez, Lorna M. Fitzpatrick, James R. Whiteford, Peter J. King, Juan P. Bolanos, Michael R. Duchen, Simon N. Waddington, Tristan R. McKay

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Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders by Leandro R Soria, Sonam Gurung, Giulia De Sabbata, Dany P Perocheau, Angela De Angelis, Gemma Bruno, Elena Polishchuk, Debora Paris, Paola Cuomo, Andrea Motta, Michael Orford, Youssef Khalil, Simon Eaton, Philippa B Mills, Simon N Waddington, Carmine Settembre, Andrés F Muro, Julien Baruteau, Nicola Brunetti‐Pierri

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Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Us... by Panicos Shangaris, Stavros P. Loukogeorgakis, Sindhu Subramaniam, Christina Flouri, Laurence H. Jackson, Wei Wang, Michael P. Blundell, Shanrun Liu, Simon Eaton, Nahla Bakhamis, Durrgah Latchumi Ramachandra, Panayiotis Maghsoudlou, Luca Urbani, Simon N. Waddington, Ayad Eddaoudi, Joy Archer, Michael N. Antoniou, Daniel J. Stuckey, Manfred Schmidt, Adrian J. Thrasher, Thomas M. Ryan, Paolo De Coppi, Anna L. David

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Regulation of post-Golgi LH3 trafficking is essential for collagen homeostasis by Blerida Banushi, Federico Forneris, Anna Straatman-Iwanowska, Adam Strange, Anne-Marie Lyne, Clare Rogerson, Jemima J. Burden, Wendy E. Heywood, Joanna Hanley, Ivan Doykov, Kornelis R. Straatman, Holly Smith, Danai Bem, Janos Kriston-Vizi, Gema Ariceta, Maija Risteli, Chunguang Wang, Rosalyn E. Ardill, Marcin Zaniew, Julita Latka-Grot, Simon N. Waddington, S. J. Howe, Francesco Ferraro, Asllan Gjinovci, Scott Lawrence, Mark Marsh, Mark Girolami, Laurent Bozec, Kevin Mills, Paul Gissen

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