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

A bioinformatic pipeline for simulating viral integration data

by Suzanne Scott, Susanna Grigson, Felix Hartkopf, Claus V. Hallwirth, Ian E. Alexander, Denis C. Bauer, Laurence O.W. Wilson
Published 2022-06-01

Performance of Cardiotropic rAAV Vectors Is Dependent on Production Method

by Renuka Rao, Melad Farraha, Grant J. Logan, Sindhu Igoor, Cindy Y. Kok, James J. H. Chong, Ian E. Alexander, Eddy Kizana
Published 2022-07-01

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

AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia

by Lara E. Graves, Eva B. van Dijk, Erhua Zhu, Sundar Koyyalamudi, Tiffany Wotton, Dinah Sung, Shubha Srinivasan, Samantha L. Ginn, Ian E. Alexander
Published 2024-06-01

Angiotensin Converting Enzyme‐2 Therapy Improves Liver Fibrosis and Glycemic Control in Diabetic Mice With Fatty Liver

by Indu G. Rajapaksha, Lakmie S. Gunarathne, Khashayar Asadi, Ross Laybutt, Sof Andrikopoulous, Ian E. Alexander, Mathew J. Watt, Peter W. Angus, Chandana B. Herath
Published 2022-05-01

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

Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice

by Sereina Deplazes, Andrea Schlegel, Zhuolun Song, Gabriella Allegri, Nicole Rimann, Tanja Scherer, Melanie Willimann, Lennart Opitz, Sharon C. Cunningham, Ian E. Alexander, Anja Kipar, Johannes Häberle, Beat Thöny, Hiu Man Grisch-Chan
Published 2022-12-01

Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy

by Arlene M. D'Silva, Sandra Holland, Didu Kariyawasam, Karen Herbert, Peter Barclay, Anita Cairns, Suzanna C. MacLennan, Monique M. Ryan, Hugo Sampaio, Nicholas Smith, Ian R. Woodcock, Eppie M. Yiu, Ian E. Alexander, Michelle A. Farrar
Published 2022-03-01

Novel AAV variants with improved tropism for human Schwann cells

by Matthieu Drouyer, Tak-Ho Chu, Elodie Labit, Florencia Haase, Renina Gale Navarro, Deborah Nazareth, Nicole Rosin, Jessica Merjane, Suzanne Scott, Marti Cabanes-Creus, Adrian Westhaus, Erhua Zhu, Rajiv Midha, Ian E. Alexander, Jeff Biernaskie, Samantha L. Ginn, Leszek Lisowski
Published 2024-06-01

Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8

by Marti Cabanes-Creus, Renina Gale Navarro, Erhua Zhu, Grober Baltazar, Sophia H.Y. Liao, Matthieu Drouyer, Anais K. Amaya, Suzanne Scott, Loan Hanh Nguyen, Adrian Westhaus, Matthias Hebben, Laurence O.W. Wilson, Adrian J. Thrasher, Ian E. Alexander, Leszek Lisowski
Published 2022-03-01

Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer’s disease

by Roy Chun-Laam Ng, Min Jian, Oscar Ka-Fai Ma, Ariya Weiman Xiang, Myriam Bunting, Jason Shing-Cheong Kwan, Curtis Wai-Kin Wong, Leung-Wah Yick, Sookja Kim Chung, Karen Siu-Ling Lam, Ian E. Alexander, Aimin Xu, Koon-Ho Chan
Published 2024-03-01

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

Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution

by Marti Cabanes-Creus, Renina Gale Navarro, Sophia H.Y. Liao, Suzanne Scott, Rodrigo Carlessi, Ramon Roca-Pinilla, Maddison Knight, Grober Baltazar, Erhua Zhu, Matthew Jones, Elena Denisenko, Alistair R.R. Forrest, Ian E. Alexander, Janina E.E. Tirnitz-Parker, Leszek Lisowski
Published 2023-03-01

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

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

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