Ub-ISAP: a streamlined UNIX pipeline for mining unique viral vector integration sites from next generation sequencing data

by Atul Kamboj, Claus V. Hallwirth, Ian E. Alexander, Geoffrey B. McCowage, Belinda Kramer
Published 2017-06-01

Liver‐Targeted Angiotensin Converting Enzyme 2 Therapy Inhibits Chronic Biliary Fibrosis in Multiple Drug‐Resistant Gene 2‐Knockout Mice

by Indu G. Rajapaksha, Lakmie S. Gunarathne, Khashayar Asadi, Sharon C. Cunningham, Alexandra Sharland, Ian E. Alexander, Peter W. Angus, Chandana B. Herath
Published 2019-12-01

Use of a Hybrid Adeno-Associated Viral Vector Transposon System to Deliver the Insulin Gene to Diabetic NOD Mice

by Que T. La, Binhai Ren, Grant J. Logan, Sharon C. Cunningham, Neeta Khandekar, Najah T. Nassif, Bronwyn A. O’Brien, Ian E. Alexander, Ann M. Simpson
Published 2020-10-01

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
Published 2021-12-01

Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids

by Marti Cabanes-Creus, Renina Gale Navarro, Sophia H.Y. Liao, Grober Baltazar, Matthieu Drouyer, Erhua Zhu, Suzanne Scott, Clement Luong, Laurence O.W. Wilson, Ian E. Alexander, Leszek Lisowski
Published 2021-06-01

Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

by Samantha L. Ginn, Anais K. Amaya, Sophia H.Y. Liao, Erhua Zhu, Sharon C. Cunningham, Michael Lee, Claus V. Hallwirth, Grant J. Logan, Szun S. Tay, Anthony J. Cesare, Hilda A. Pickett, Markus Grompe, Kimberley Dilworth, Leszek Lisowski, Ian E. Alexander
Published 2020-02-01

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

by Marti Cabanes-Creus, Samantha L. Ginn, Anais K. Amaya, Sophia H.Y. Liao, Adrian Westhaus, Claus V. Hallwirth, Patrick Wilmott, Jason Ward, Kimberley L. Dilworth, Giorgia Santilli, Arkadiusz Rybicki, Hiroyuki Nakai, Adrian J. Thrasher, Adrian C. Filip, Ian E. Alexander, Leszek Lisowski
Published 2019-03-01

Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency

by Samantha L. Ginn, Claus V. Hallwirth, Sophia H.Y. Liao, Erdahl T. Teber, Jonathan W. Arthur, Jianmin Wu, Hong Ching Lee, Szun S. Tay, Min Hu, Roger R. Reddel, Matthew P. McCormack, Adrian J. Thrasher, Marina Cavazzana, Stephen I. Alexander, Ian E. Alexander
Published 2017-03-01

Coherence analysis discriminates between retroviral integration patterns in CD34+ cells transduced under differing clinical trial conditions

by Claus V Hallwirth, Gagan Garg, Timothy J Peters, Belinda A Kramer, Nirav V Malani, Jessica Hyman, Xiaoan Ruan, Samantha L Ginn, Nicola A Hetherington, Lavanya Veeravalli, Atif Shahab, Shoba Ranganathan, Chia-Lin Wei, Christopher Liddle, Adrian J Thrasher, Frederic D Bushman, Michael J Buckley, Ian E Alexander
Published 2015-01-01

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2

by Marti Cabanes-Creus, Adrian Westhaus, Renina Gale Navarro, Grober Baltazar, Erhua Zhu, Anais K. Amaya, Sophia H.Y. Liao, Suzanne Scott, Erwan Sallard, Kimberley L. Dilworth, Arkadiusz Rybicki, Matthieu Drouyer, Claus V. Hallwirth, Antonette Bennett, Giorgia Santilli, Adrian J. Thrasher, Mavis Agbandje-McKenna, Ian E. Alexander, Leszek Lisowski
Published 2020-06-01