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Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

Author

Listed:
  • Kosuke Yusa

    (Wellcome Trust Sanger Institute, Wellcome Trust Genome Campus, Hinxton)

  • S. Tamir Rashid

    (Anne McLaren Laboratory for Regenerative Medicine, West Forvie Building, Robinson Way, University of Cambridge
    University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building)

  • Helene Strick-Marchand

    (Innate Immunity Unit, Institut Pasteur
    INSERM, U668)

  • Ignacio Varela

    (Instituto de Biomedicina y Biotecnología de Cantabria (IBBTEC), CSIC-UC-SODERCAN Avda. Cardenal Herrera Oria s/n 39011 Santander)

  • Pei-Qi Liu

    (Sangamo BioSciences Inc.)

  • David E. Paschon

    (Sangamo BioSciences Inc.)

  • Elena Miranda

    (University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building
    Dept. Biologia e Biotecnologie ‘Charles Darwin’, Università di Roma “La Sapienza”, p.le Aldo Moro 5)

  • Adriana Ordóñez

    (University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building)

  • Nicholas R. F. Hannan

    (Anne McLaren Laboratory for Regenerative Medicine, West Forvie Building, Robinson Way, University of Cambridge)

  • Foad J. Rouhani

    (Wellcome Trust Sanger Institute, Wellcome Trust Genome Campus, Hinxton
    Anne McLaren Laboratory for Regenerative Medicine, West Forvie Building, Robinson Way, University of Cambridge)

  • Sylvie Darche

    (Innate Immunity Unit, Institut Pasteur
    INSERM, U668)

  • Graeme Alexander

    (Cambridge University Hospitals NHS Trust)

  • Stefan J. Marciniak

    (University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building)

  • Noemi Fusaki

    (DNAVEC Corporation, Tsukuba
    PRESTO, JST)

  • Mamoru Hasegawa

    (DNAVEC Corporation, Tsukuba)

  • Michael C. Holmes

    (Sangamo BioSciences Inc.)

  • James P. Di Santo

    (Innate Immunity Unit, Institut Pasteur
    INSERM, U668)

  • David A. Lomas

    (University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building)

  • Allan Bradley

    (Wellcome Trust Sanger Institute, Wellcome Trust Genome Campus, Hinxton)

  • Ludovic Vallier

    (Anne McLaren Laboratory for Regenerative Medicine, West Forvie Building, Robinson Way, University of Cambridge)

Abstract

Fixing the genes in iPS cells Before human induced pluripotent stem (iPS) cells can be used to treat genetically inherited human disease, it will be necessary to develop methods of correcting disease-causing mutations that are compatible with clinical applications, combining efficiency with efficacy and leaving no residual sequences in the targeted genome. Yusa et al. present a proof-of-principle experiment demonstrating the complete genetic correction of a disease-causing mutation in patient-specific iPS cells. They use zinc finger nucleases and piggyBac technology to correction a point mutation in the α1-antitrypsin gene, which is responsible for α1-antitrypsin deficiency (A1ATD). The corrected iPS cells could efficiently differentiate to form hepatocyte-like cells and engraft into an animal model for liver injury without tumour formation.

Suggested Citation

  • Kosuke Yusa & S. Tamir Rashid & Helene Strick-Marchand & Ignacio Varela & Pei-Qi Liu & David E. Paschon & Elena Miranda & Adriana Ordóñez & Nicholas R. F. Hannan & Foad J. Rouhani & Sylvie Darche & Gr, 2011. "Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells," Nature, Nature, vol. 478(7369), pages 391-394, October.
    • Handle: RePEc:nat:nature:v:478:y:2011:i:7369:d:10.1038_nature10424
    DOI: 10.1038/nature10424
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