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Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells.
Crane, Ana M; Kramer, Philipp; Bui, Jacquelin H; Chung, Wook Joon; Li, Xuan Shirley; Gonzalez-Garay, Manuel L; Hawkins, Finn; Liao, Wei; Mora, Daniela; Choi, Sangbum; Wang, Jianbin; Sun, Helena C; Paschon, David E; Guschin, Dmitry Y; Gregory, Philip D; Kotton, Darrell N; Holmes, Michael C; Sorscher, Eric J; Davis, Brian R.
Afiliación
  • Crane AM; Center for Stem Cell and Regenerative Medicine, Brown Foundation Institute of Molecular Medicine, University of Texas Health Science Center, Houston, TX 77030, USA.
  • Kramer P; Center for Stem Cell and Regenerative Medicine, Brown Foundation Institute of Molecular Medicine, University of Texas Health Science Center, Houston, TX 77030, USA.
  • Bui JH; Center for Stem Cell and Regenerative Medicine, Brown Foundation Institute of Molecular Medicine, University of Texas Health Science Center, Houston, TX 77030, USA.
  • Chung WJ; Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama, Birmingham, AL 35294, USA.
  • Li XS; Center for Stem Cell and Regenerative Medicine, Brown Foundation Institute of Molecular Medicine, University of Texas Health Science Center, Houston, TX 77030, USA.
  • Gonzalez-Garay ML; Center for Molecular Imaging, Brown Foundation Institute of Molecular Medicine, University of Texas Health Science Center, Houston, TX 77030, USA.
  • Hawkins F; Center for Regenerative Medicine, Boston University and Boston Medical Center, Boston, MA 02118, USA.
  • Liao W; Center for Stem Cell and Regenerative Medicine, Brown Foundation Institute of Molecular Medicine, University of Texas Health Science Center, Houston, TX 77030, USA.
  • Mora D; Center for Stem Cell and Regenerative Medicine, Brown Foundation Institute of Molecular Medicine, University of Texas Health Science Center, Houston, TX 77030, USA.
  • Choi S; Division of Clinical and Translational Sciences, Department of Internal Medicine, University of Texas Health Science Center, Houston, TX 77030, USA.
  • Wang J; Sangamo BioSciences, Inc., Richmond, CA 94804, USA.
  • Sun HC; Sangamo BioSciences, Inc., Richmond, CA 94804, USA.
  • Paschon DE; Sangamo BioSciences, Inc., Richmond, CA 94804, USA.
  • Guschin DY; Sangamo BioSciences, Inc., Richmond, CA 94804, USA.
  • Gregory PD; Sangamo BioSciences, Inc., Richmond, CA 94804, USA.
  • Kotton DN; Center for Regenerative Medicine, Boston University and Boston Medical Center, Boston, MA 02118, USA.
  • Holmes MC; Sangamo BioSciences, Inc., Richmond, CA 94804, USA.
  • Sorscher EJ; Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama, Birmingham, AL 35294, USA.
  • Davis BR; Center for Stem Cell and Regenerative Medicine, Brown Foundation Institute of Molecular Medicine, University of Texas Health Science Center, Houston, TX 77030, USA. Electronic address: brian.r.davis@uth.tmc.edu.
Stem Cell Reports ; 4(4): 569-77, 2015 Apr 14.
Article en En | MEDLINE | ID: mdl-25772471
Recently developed reprogramming and genome editing technologies make possible the derivation of corrected patient-specific pluripotent stem cell sources-potentially useful for the development of new therapeutic approaches. Starting with skin fibroblasts from patients diagnosed with cystic fibrosis, we derived and characterized induced pluripotent stem cell (iPSC) lines. We then utilized zinc-finger nucleases (ZFNs), designed to target the endogenous CFTR gene, to mediate correction of the inherited genetic mutation in these patient-derived lines via homology-directed repair (HDR). We observed an exquisitely sensitive, homology-dependent preference for targeting one CFTR allele versus the other. The corrected cystic fibrosis iPSCs, when induced to differentiate in vitro, expressed the corrected CFTR gene; importantly, CFTR correction resulted in restored expression of the mature CFTR glycoprotein and restoration of CFTR chloride channel function in iPSC-derived epithelial cells.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Marcación de Gen / Regulador de Conductancia de Transmembrana de Fibrosis Quística / Fibrosis Quística / Células Madre Pluripotentes Inducidas Límite: Humans Idioma: En Revista: Stem Cell Reports Año: 2015 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Marcación de Gen / Regulador de Conductancia de Transmembrana de Fibrosis Quística / Fibrosis Quística / Células Madre Pluripotentes Inducidas Límite: Humans Idioma: En Revista: Stem Cell Reports Año: 2015 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos