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Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations

Cell, 2011-07, Vol.146 (2), p.318-331 [Peer Reviewed Journal]

2011 Elsevier Inc. ;Copyright © 2011 Elsevier Inc. All rights reserved. ;2011 Elsevier Inc. All rights reserved. 2011 ;ISSN: 0092-8674 ;EISSN: 1097-4172 ;DOI: 10.1016/j.cell.2011.06.019 ;PMID: 21757228

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Title:
Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations
Author: Soldner, Frank ; Laganière, Josée ; Cheng, Albert W. ; Hockemeyer, Dirk ; Gao, Qing ; Alagappan, Raaji ; Khurana, Vikram ; Golbe, Lawrence I. ; Myers, Richard H. ; Lindquist, Susan ; Zhang, Lei ; Guschin, Dmitry ; Fong, Lauren K. ; Vu, B. Joseph ; Meng, Xiangdong ; Urnov, Fyodor D. ; Rebar, Edward J. ; Gregory, Philip D. ; Zhang, H. Steve ; Jaenisch, Rudolf
Subjects: Age ; alpha-Synuclein - genetics ; biomedical research ; Cell Line ; disease control ; diseases ; Embryonic Stem Cells ; genes ; genetic background ; Genetic Engineering ; Genome-Wide Association Study ; Genomes ; human diseases ; Humans ; induced pluripotent stem cells ; Inhibitory postsynaptic potentials ; Movement disorders ; Mutagenesis ; mutation ; Neurodegenerative diseases ; Nuclease ; Oligonucleotides - metabolism ; Parkinson disease ; Parkinson Disease - pathology ; Parkinson's disease ; phenotype ; Pluripotent Stem Cells ; Point Mutation ; Somatic cells ; Stem cells ; Synuclein ; zinc finger motif ; Zinc finger proteins
Is Part Of: Cell, 2011-07, Vol.146 (2), p.318-331
Description: Patient-specific induced pluripotent stem cells (iPSCs) derived from somatic cells provide a unique tool for the study of human disease, as well as a promising source for cell replacement therapies. One crucial limitation has been the inability to perform experiments under genetically defined conditions. This is particularly relevant for late age onset disorders in which in vitro phenotypes are predicted to be subtle and susceptible to significant effects of genetic background variations. By combining zinc finger nuclease (ZFN)-mediated genome editing and iPSC technology, we provide a generally applicable solution to this problem, generating sets of isogenic disease and control human pluripotent stem cells that differ exclusively at either of two susceptibility variants for Parkinson's disease by modifying the underlying point mutations in the α-synuclein gene. The robust capability to genetically correct disease-causing point mutations in patient-derived hiPSCs represents significant progress for basic biomedical research and an advance toward hiPSC-based cell replacement therapies. [Display omitted] ► General approach to generate isogenic disease and control pluripotent stem cells ► ZFNs-mediated genome editing of human pluripotent stem cells without drug selection ► Generation of isogenic cells differing at two PD-causing mutations in α-synuclein ► Correction of PD-causing A53T mutation in patient-derived hiPSCs
Publisher: United States: Elsevier Inc
Language: English
Identifier: ISSN: 0092-8674
EISSN: 1097-4172
DOI: 10.1016/j.cell.2011.06.019
PMID: 21757228
Source: MEDLINE
Open Access: Cell Press Free Archives

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Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations

2011 Elsevier Inc. ;Copyright © 2011 Elsevier Inc. All rights reserved. ;2011 Elsevier Inc. All rights reserved. 2011 ;ISSN: 0092-8674 ;EISSN: 1097-4172 ;DOI: 10.1016/j.cell.2011.06.019 ;PMID: 21757228

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