Research ArticleGene Therapy

Restoring the natural tropism of AAV2 vectors for human liver

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Science Translational Medicine  09 Sep 2020:
Vol. 12, Issue 560, eaba3312
DOI: 10.1126/scitranslmed.aba3312

Article Information

vol. 12 no. 560

PubMed: 
Print ISSN: 
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History: 
  • Received for publication December 3, 2019
  • Resubmitted March 25, 2020
  • Accepted for publication August 20, 2020
  • .

Author Information

  1. Marti Cabanes-Creus1,
  2. Claus V. Hallwirth2,
  3. Adrian Westhaus1,3,
  4. Boaz H. Ng4,
  5. Sophia H.Y. Liao1,
  6. Erhua Zhu2,
  7. Renina Gale Navarro1,
  8. Grober Baltazar1,
  9. Matthieu Drouyer1,
  10. Suzanne Scott2,5,
  11. Grant J. Logan2,
  12. Giorgia Santilli3,
  13. Antonette Bennett6,
  14. Samantha L. Ginn2,
  15. Geoff McCaughan7,
  16. Adrian J. Thrasher3,
  17. Mavis Agbandje-McKenna6,
  18. Ian E. Alexander2,8 and
  19. Leszek Lisowski1,4,9,*
  1. 1Translational Vectorology Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
  2. 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.
  3. 3Great Ormond Institute of Child Health, University College London, WC1N 1EH London, UK.
  4. 4Vector and Genome Engineering Facility, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
  5. 5Commonwealth Scientific and Industrial Research Organisation (CSIRO), North Ryde, NSW 2113, Australia.
  6. 6Department of Biochemistry and Molecular Biology, Center for Structural Biology, University of Florida, Gainesville, FL 32610, USA.
  7. 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.
  8. 8Discipline of Child and Adolescent Health, The University of Sydney, Sydney Medical School, Faculty of Medicine and Health, Westmead, NSW 2145, Australia.
  9. 9Military Institute of Hygiene and Epidemiology, Biological Threats Identification and Countermeasure Centre, 24-100 Puławy, Poland.
  1. *Corresponding author. Email: llisowski{at}cmri.org.au

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