Restoring the natural tropism of AAV2 vectors for human liver

Marti Cabanes-Creus; Claus V. Hallwirth; Adrian Westhaus; Boaz H. Ng; Sophia H.Y. Liao; Erhua Zhu; Renina Gale Navarro; Grober Baltazar; Matthieu Drouyer; Suzanne Scott; Grant J. Logan; Giorgia Santilli; Antonette Bennett; Samantha L. Ginn; Geoff McCaughan; Adrian J. Thrasher; Mavis Agbandje-McKenna; Ian E. Alexander; Leszek Lisowski

doi:10.1126/scitranslmed.aba3312

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 Bennett ⁶,
View ORCID ProfileSamantha L. Ginn²,
View ORCID ProfileGeoff McCaughan⁷,
View ORCID ProfileAdrian J. Thrasher³,
View ORCID ProfileMavis Agbandje-McKenna ⁶,
View ORCID ProfileIan E. Alexander ²,⁸ and
View ORCID ProfileLeszek Lisowski ¹,⁴,⁹,*

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

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.

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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.

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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.

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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.

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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.

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Erhua Zhu

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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.

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Grober Baltazar

1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.

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Matthieu Drouyer

1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.

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Suzanne Scott

5Commonwealth Scientific and Industrial Research Organisation (CSIRO), North Ryde, NSW 2113, Australia.

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Grant J. Logan

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Giorgia Santilli

3Great Ormond Institute of Child Health, University College London, WC1N 1EH London, UK.

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Antonette Bennett

6Department of Biochemistry and Molecular Biology, Center for Structural Biology, University of Florida, Gainesville, FL 32610, USA.

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Samantha L. Ginn

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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.

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Adrian J. Thrasher

3Great Ormond Institute of Child Health, University College London, WC1N 1EH London, UK.

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Mavis Agbandje-McKenna

6Department of Biochemistry and Molecular Biology, Center for Structural Biology, University of Florida, Gainesville, FL 32610, USA.

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Ian E. Alexander

8Discipline of Child and Adolescent Health, The University of Sydney, Sydney Medical School, Faculty of Medicine and Health, Westmead, NSW 2145, Australia.

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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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For correspondence: llisowski@cmri.org.au

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