Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors

Author:

Westhaus Adrian12ORCID,Cabanes-Creus Marti1,Dilworth Kimberley L.1,Zhu Erhua3,Salas Gómez David4,Navarro Renina G.1,Amaya Anais K.3ORCID,Scott Suzanne135,Kwiatek Magdalena6,McCorkindale Alexandra L.7,Hayman Tara E.7,Frahm Silke8,Perocheau Dany P.91011,Tran Bang Manh12,Vincan Elizabeth121314,Wong Sharon L.1516,Waters Shafagh A.151617,Riddiough Georgina E.1218,Perini Marcos V.18,Wilson Laurence O.W.519,Baruteau Julien91011,Diecke Sebastian8,González-Aseguinolaza Gloria4ORCID,Santilli Giorgia2ORCID,Thrasher Adrian J.2ORCID,Alexander Ian E.320,Lisowski Leszek12122ORCID

Affiliation:

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

2. Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

3. Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia

4. Gene Therapy and Regulation of Gene Expression Department, IdiSNA, Instituto de Investigación Sanitaria de Navarra, Universidad de Navarra, CIMA, Pamplona, Spain

5. Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation, Sydney, Australia

6. Military Institute of Hygiene and Epidemiology, The Biological Threats Identification and Countermeasure Centre, Puławy, Poland

7. Inventia Life Science Pty Ltd., Sydney, Australia

8. Stem Cell Technology Platform, Max Delbrück Centrum for Molecular Medicine, Berlin, Germany

9. Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

10. Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom

11. National Institute of Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom

12. Department of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia

13. Victorian Infectious Diseases Reference Laboratory, Royal Melbourne Hospital at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia

14. Curtin Medical School, Curtin University, Perth, Australia

15. Molecular and Integrative Cystic Fibrosis (miCF) Research Centre, University of New South Wales and Sydney Children's Hospital, Sydney, Australia

16. School of Biomedical Sciences, Faculty of Medicine, University of New South Wales, Sydney, Australia

17. Department of Respiratory Medicine, Sydney Children's Hospital, Faculty of Medicine, University of New South Wales, Sydney, Australia

18. Department of Surgery, Austin Health Precinct, The University of Melbourne, Austin Health, Heidelberg, Australia

19. Applied BioSciences, Faculty of Science and Engineering, Macquarie University, Sydney, Australia

20. Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, Australia

21. Vector and Genome Engineering Facility, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia

22. Laboratory of Molecular Oncology and Innovative Therapies, Military Institute of Medicine, Warszawa, Poland.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

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