Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations
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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.
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CRISPR-induced DNA breaks are especially toxic to iPSCs, lowering survival and potentially confounding observed phenotypes.
“However, CRISPR-induced DNA breaks are particularly toxic to iPSCs111, reducing cell survival and potentially confounding the observed phenotypes.”
From CRISPR-based functional genomics for neurological disease (2020) · cites this paper as [111]
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- Authors
- Soldner, F., Laganière, J., Cheng, A. W., Hockemeyer, D., Gao, Q., Alagappan, R., Khurana, V., Golbe, L. I., Myers, R. H., Lindquist, S., Zhang, L., Guschin, D., Fong, L. K., Vu, B. J., Meng, X., Urnov, F. D., Rebar, E. J., Gregory, P. D., Zhang, H. S., & Jaenisch, R.
- Affiliations
- Whitehead Institute for Biomedical Research (US)
- Sangamo BioSciences (United States) (US)
- Brigham and Women's Hospital (US)
- Rutgers, The State University of New Jersey (US)
- Boston University (US)
- Corresponding author
- Jaenisch, Rudolf
- ORCID
- Venue
- Cell
- Publisher
- Elsevier BV
- Published
- 2011-07
- Volume
- 146
- Issue
- 2
- Pages
- 318-331
- ISSN
- 0092-8674 (ISSN-L)
- PMC
- PMC3155290
- OpenAlex
- W2154679217
- Cited by
- 728
- References
- 58
- Citations by year
- 2026: 7 · 2025: 16 · 2024: 21 · 2023: 24 · 2022: 21 · 2021: 36 · 2020: 29 · 2019: 36 · 2018: 52 · 2017: 48 · 2016: 69 · 2015: 83 · 2014: 92 · 2013: 77 · 2012: 86
- FWCI
- 36.97
- Type
- journal article
- Language
- en
- Update notices
- erratum (2011-08-19)
- Open access
- bronze
- doi.org
- Licence
- Locations
- Cell (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- DSpace@MIT (Massachusetts Institute of Technology) (submittedVersion, cc-by-nc)
- Funders
- Howard Hughes Medical Institute
- Life Sciences Research Foundation
- National Institutes of Health (2r01ca084198-10, HD 045022)
- National Cancer Institute (R01-CA084198)
- National Institute of Neurological Disorders and Stroke (R01NS076843)
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (R01HD045022, R37HD045022)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Pluripotent Stem Cells Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Health Sciences › Medicine › Neurology › Parkinson's Disease Mechanisms and Treatments
- Keywords
- Parkinson's disease, induced pluripotent stem cells, alpha-synuclein, point mutations, zinc finger nucleases, genome editing, gene correction, disease modeling, cell replacement therapy, genetic background
- MeSH terms
- Cell Line, Genetic Engineering, Humans, Oligonucleotides, Parkinson Disease, Mutagenesis, Point Mutation, Pluripotent Stem Cells, alpha-Synuclein, Embryonic Stem Cells, Genome-Wide Association Study
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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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