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

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, Graeme Alexander, Stefan J. Marciniak, Noemi Fusaki, Mamoru Hasegawa, Michael C. Holmes, James P. Di Santo, David A. Lomas, Allan Bradley () and Ludovic Vallier ()
Additional contact information
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
Helene Strick-Marchand: Innate Immunity Unit, Institut Pasteur
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
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
Sylvie Darche: Innate Immunity Unit, Institut Pasteur
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
Mamoru Hasegawa: DNAVEC Corporation, Tsukuba
Michael C. Holmes: Sangamo BioSciences Inc.
James P. Di Santo: Innate Immunity Unit, Institut Pasteur
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

Nature, 2011, vol. 478, issue 7369, 391-394

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.

Date: 2011
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DOI: 10.1038/nature10424

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