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

Author

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  • Leszek Lisowski

    (Stanford University, School of Medicine, 269 Campus Drive
    Present addresses: Gene Transfer, Targeting and Therapeutics Core, The Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd, San Diego, California 92037, USA (L.L.); Department of Haematology, University College London Cancer Institute, London WC1E 6BT, UK (A.P.D.).)

  • 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
    Present addresses: Gene Transfer, Targeting and Therapeutics Core, The Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd, San Diego, California 92037, USA (L.L.); Department of Haematology, University College London Cancer Institute, London WC1E 6BT, UK (A.P.D.).)

  • 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
    Discipline of Paediatrics and Child Health, The University of Sydney, 2145 New South Wales, Australia)

  • Mark A. Kay

    (Stanford University, School of Medicine, 269 Campus Drive)

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.

Suggested Citation

  • Leszek Lisowski & Allison P. Dane & Kirk Chu & Yue Zhang & Sharon C. Cunningham & Elizabeth M. Wilson & Sean Nygaard & Markus Grompe & Ian E. Alexander & Mark A. Kay, 2014. "Selection and evaluation of clinically relevant AAV variants in a xenograft liver model," Nature, Nature, vol. 506(7488), pages 382-386, February.
    • Handle: RePEc:nat:nature:v:506:y:2014:i:7488:d:10.1038_nature12875
    DOI: 10.1038/nature12875
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    Cited by:

    1. Adriana Gonzalez-Sandoval & Katja Pekrun & Shinnosuke Tsuji & Feijie Zhang & King L. Hung & Howard Y. Chang & Mark A. Kay, 2023. "The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner," Nature Communications, Nature, vol. 14(1), pages 1-11, December.
    2. Clément Pontoizeau & Marcelo Simon-Sola & Clovis Gaborit & Vincent Nguyen & Irina Rotaru & Nolan Tual & Pasqualina Colella & Muriel Girard & Maria-Grazia Biferi & Jean-Baptiste Arnoux & Agnès Rötig & , 2022. "Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice," Nature Communications, Nature, vol. 13(1), pages 1-13, December.
    3. Mercedes Barzi & Tong Chen & Trevor J. Gonzalez & Francis P. Pankowicz & Seh Hoon Oh & Helen L. Streff & Alan Rosales & Yunhan Ma & Sabrina Collias & Sarah E. Woodfield & Anna Mae Diehl & Sanjeev A. V, 2024. "A humanized mouse model for adeno-associated viral gene therapy," Nature Communications, Nature, vol. 15(1), pages 1-10, December.
    4. Helena Costa-Verdera & Fanny Collaud & Christopher R. Riling & Pauline Sellier & Jayme M. L. Nordin & G. Michael Preston & Umut Cagin & Julien Fabregue & Simon Barral & Maryse Moya-Nilges & Jacomina K, 2021. "Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates," Nature Communications, Nature, vol. 12(1), pages 1-16, December.

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