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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

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Listed:
  • A. Barzel

    (269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA)

  • N. K. Paulk

    (269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA)

  • Y. Shi

    (Box 1270, UCSF, San Francisco, California 94143-1270, USA)

  • Y. Huang

    (269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA)

  • K. Chu

    (269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA)

  • F. Zhang

    (269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA)

  • P. N. Valdmanis

    (269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA)

  • L. P. Spector

    (269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA)

  • M. H. Porteus

    (269 Campus Drive, Lorry Lokey Stem Cell Research Building, Room G3045, Stanford, California 94305-5164, USA)

  • K. M. Gaensler

    (Box 1270, UCSF, San Francisco, California 94143-1270, USA)

  • M. A. Kay

    (269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA)

Abstract

Promoterless recombinant adeno-associated virus is used without nucleases to target the human coagulation factor IX gene to the liver-expressed albumin locus in haemophilia B mice, with an on-target integration into ∼0.5% of the albumin alleles in hepatocytes; stable F9 plasma levels at 7–20% of normal were obtained, leading to normal coagulation times in treated factor-IX-deficient mice.

Suggested Citation

  • A. Barzel & N. K. Paulk & Y. Shi & Y. Huang & K. Chu & F. Zhang & P. N. Valdmanis & L. P. Spector & M. H. Porteus & K. M. Gaensler & M. A. Kay, 2015. "Promoterless gene targeting without nucleases ameliorates haemophilia B in mice," Nature, Nature, vol. 517(7534), pages 360-364, January.
    • Handle: RePEc:nat:nature:v:517:y:2015:i:7534:d:10.1038_nature13864
    DOI: 10.1038/nature13864
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    Cited by:

    1. Amita Tiyaboonchai & Anne Vonada & Jeffrey Posey & Carl Pelz & Leslie Wakefield & Markus Grompe, 2022. "Self-cleaving guide RNAs enable pharmacological selection of precise gene editing events in vivo," Nature Communications, Nature, vol. 13(1), pages 1-12, December.
    2. Patrizia Tornabene & Rita Ferla & Manel Llado-Santaeularia & Miriam Centrulo & Margherita Dell’Anno & Federica Esposito & Elena Marrocco & Emanuela Pone & Renato Minopoli & Carolina Iodice & Edoardo N, 2022. "Therapeutic homology-independent targeted integration in retina and liver," Nature Communications, Nature, vol. 13(1), pages 1-14, December.

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