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Research ArticleGene Therapy
Restoring the natural tropism of AAV2 vectors for human liver
- View ORCID ProfileMarti Cabanes-Creus1,
- View ORCID ProfileClaus V. Hallwirth2,
- Adrian Westhaus1,3,
- View ORCID ProfileBoaz H. Ng4,
- Sophia H.Y. Liao1,
- View ORCID ProfileErhua Zhu2,
- View ORCID ProfileRenina Gale Navarro1,
- View ORCID ProfileGrober Baltazar1,
- View ORCID ProfileMatthieu Drouyer1,
- View ORCID ProfileSuzanne Scott2,5,
- Grant J. Logan2,
- Giorgia Santilli3,
- View ORCID ProfileAntonette Bennett6,
- View ORCID ProfileSamantha L. Ginn2,
- View ORCID ProfileGeoff McCaughan7,
- View ORCID ProfileAdrian J. Thrasher3,
- View ORCID ProfileMavis Agbandje-McKenna6,
- View ORCID ProfileIan E. Alexander2,8 and
- View ORCID ProfileLeszek Lisowski1,4,9,*
- 1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
- 2Gene Therapy Research Unit, Children’s Medical Research Institute and Children’s Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW 2145, Australia.
- 3Great Ormond Institute of Child Health, University College London, WC1N 1EH London, UK.
- 4Vector and Genome Engineering Facility, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
- 5Commonwealth Scientific and Industrial Research Organisation (CSIRO), North Ryde, NSW 2113, Australia.
- 6Department of Biochemistry and Molecular Biology, Center for Structural Biology, University of Florida, Gainesville, FL 32610, USA.
- 7Liver Injury and Cancer Program, Centenary Research Institute, A.W Morrow Gastroenterology and Liver Centre, Australian National Liver Transplant Unit, Royal Prince Alfred Hospital, The University of Sydney, Sydney, NSW 2006, Australia.
- 8Discipline of Child and Adolescent Health, The University of Sydney, Sydney Medical School, Faculty of Medicine and Health, Westmead, NSW 2145, Australia.
- 9Military Institute of Hygiene and Epidemiology, Biological Threats Identification and Countermeasure Centre, 24-100 Puławy, Poland.
- ↵*Corresponding author. Email: llisowski{at}cmri.org.au
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Science Translational Medicine 09 Sep 2020:
Vol. 12, Issue 560, eaba3312
DOI: 10.1126/scitranslmed.aba3312
Vol. 12, Issue 560, eaba3312
DOI: 10.1126/scitranslmed.aba3312
Marti Cabanes-Creus
1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Claus V. Hallwirth
2Gene Therapy Research Unit, Children’s Medical Research Institute and Children’s Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW 2145, Australia.
Adrian Westhaus
1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
3Great Ormond Institute of Child Health, University College London, WC1N 1EH London, UK.
Boaz H. Ng
4Vector and Genome Engineering Facility, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Sophia H.Y. Liao
1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Erhua Zhu
2Gene Therapy Research Unit, Children’s Medical Research Institute and Children’s Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW 2145, Australia.
Renina Gale Navarro
1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Grober Baltazar
1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Matthieu Drouyer
1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Suzanne Scott
2Gene Therapy Research Unit, Children’s Medical Research Institute and Children’s Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW 2145, Australia.
5Commonwealth Scientific and Industrial Research Organisation (CSIRO), North Ryde, NSW 2113, Australia.
Grant J. Logan
2Gene Therapy Research Unit, Children’s Medical Research Institute and Children’s Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW 2145, Australia.
Giorgia Santilli
3Great Ormond Institute of Child Health, University College London, WC1N 1EH London, UK.
Antonette Bennett
6Department of Biochemistry and Molecular Biology, Center for Structural Biology, University of Florida, Gainesville, FL 32610, USA.
Samantha L. Ginn
2Gene Therapy Research Unit, Children’s Medical Research Institute and Children’s Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW 2145, Australia.
Geoff McCaughan
7Liver Injury and Cancer Program, Centenary Research Institute, A.W Morrow Gastroenterology and Liver Centre, Australian National Liver Transplant Unit, Royal Prince Alfred Hospital, The University of Sydney, Sydney, NSW 2006, Australia.
Adrian J. Thrasher
3Great Ormond Institute of Child Health, University College London, WC1N 1EH London, UK.
Mavis Agbandje-McKenna
6Department of Biochemistry and Molecular Biology, Center for Structural Biology, University of Florida, Gainesville, FL 32610, USA.
Ian E. Alexander
2Gene Therapy Research Unit, Children’s Medical Research Institute and Children’s Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW 2145, Australia.
8Discipline of Child and Adolescent Health, The University of Sydney, Sydney Medical School, Faculty of Medicine and Health, Westmead, NSW 2145, Australia.
Leszek Lisowski
1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
4Vector and Genome Engineering Facility, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
9Military Institute of Hygiene and Epidemiology, Biological Threats Identification and Countermeasure Centre, 24-100 Puławy, Poland.
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