Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Leszek Lisowski, Allison P. Dane, Kirk Chu, Yue Zhang, Sharon C. Cunningham, Elizabeth M. Wilson, Sean Nygaard, Markus Grompe, Ian E. Alexander and Mark A. Kay ()
Additional contact information
Leszek Lisowski: Stanford University, School of Medicine, 269 Campus Drive
Allison P. Dane: Gene Therapy Research Unit, The Children's Hospital at Westmead and Children’s Medical Research Institute, Locked Bag 4001, Westmead, 2145 New South Wales, Australia
Kirk Chu: Stanford University, School of Medicine, 269 Campus Drive
Yue Zhang: Stanford University, School of Medicine, 269 Campus Drive
Sharon C. Cunningham: Gene Therapy Research Unit, The Children's Hospital at Westmead and Children’s Medical Research Institute, Locked Bag 4001, Westmead, 2145 New South Wales, Australia
Elizabeth M. Wilson: Yecuris Corporation
Sean Nygaard: Oregon Stem Cell Center, Oregon Health and Science University
Markus Grompe: Oregon Stem Cell Center, Oregon Health and Science University
Ian E. Alexander: Gene Therapy Research Unit, The Children's Hospital at Westmead and Children’s Medical Research Institute, Locked Bag 4001, Westmead, 2145 New South Wales, Australia
Mark A. Kay: Stanford University, School of Medicine, 269 Campus Drive

Nature, 2014, vol. 506, issue 7488, 382-386

Abstract: Chimaeric human–murine adeno-associated virus (AAV) capsids are described that transduce human primary hepatocytes more efficiently than currently used AAV vectors; the novel vectors may be good clinical candidates.

Date: 2014
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DOI: 10.1038/nature12875

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