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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Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice by Loukia Touramanidou, Sonam Gurung, Claudiu A. Cozmescu, Dany Perocheau, Dale Moulding, Patrick F. Finn, Andrea Frassetto, Simon N. Waddington, Paul Gissen, Julien Baruteau

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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 by Megha S. Nivsarkar, Suzanne M. K. Buckley, Alan L. Parker, Dany Perocheau, Tristan R. McKay, Ahad A. Rahim, Steven J. Howe, Simon N. Waddington

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Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model by Andrea Wetzel, Si Hang Lei, Tiansheng Liu, Michael P. Hughes, Yunan Peng, Tristan McKay, Simon N. Waddington, Simone Grannò, Ahad A. Rahim, Kirsten Harvey

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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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Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome by Filipa M Lopes, Celine Grenier, Benjamin W Jarvis, Sara Al Mahdy, Adrian Lène-McKay, Alison M Gurney, William G Newman, Simon N Waddington, Adrian S Woolf, Neil A Roberts

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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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Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice by John R. Counsell, Rajvinder Karda, Juan Antinao Diaz, Louise Carey, Tatiana Wiktorowicz, Suzanne M.K. Buckley, Shima Ameri, Joanne Ng, Julien Baruteau, Filipa Almeida, Rohan de Silva, Roberto Simone, Eleonora Lugarà, Gabriele Lignani, Dirk Lindemann, Axel Rethwilm, Ahad A. Rahim, Simon N. Waddington, Steven J. Howe

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

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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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