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Generation of an ICF Syndrome Model by Efficient Genome Editing of Human Induced Pluripotent Stem Cells Using the CRISPR System
Abstract
Genome manipulation of human induced pluripotent stem (iPS) cells is essential to achieve their full potential as tools for regenerative medicine. To date, however, gene targeting in human pluripotent stem cells (hPSCs) has proven to be extremely difficult. Recently, an efficient genome manipulation technology using the RNA-guided DNase Cas9, the clustered regularly interspaced short palindromic repeats (CRISPR) system, has been developed. Here we report the efficient generation of an iPS cell model for immunodeficiency, centromeric region instability, facial anomalies syndrome (ICF) syndrome using the CRISPR system. We obtained iPS cells with mutations in both alleles of DNA methyltransferase 3B (DNMT3B) in 63% of transfected clones. Our data suggest that the CRISPR system is highly efficient and useful for genome engineering of human iPS cells.
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“Lipofectamine has been used to deliver Cas9- and sgRNA-encoding plasmid DNA to human pluripotent stem cells to generate a model for Immunodeficiency, Centromeric region instability, Facial anomalies syndrome (ICF) syndrome with 63% transfection efficiency (Horii et al., 2013)”
From Delivering CRISPR: a review of the challenges and approaches (2018) · cites this paper as Horii et al., 2013
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“This suggests off-target mutations could be low [24].”
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Research works printed but not linked: 21
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Cited by
Sorted by citations
Genetic screens in human cells using the CRISPR-Cas9 system
Wang, T., Wei, J. J., Sabatini, D. M., & Lander, E. S. (2014) · Science · doi:10.1126/science.1246981 · Cited by 2,856
Delivering CRISPR: a review of the challenges and approaches
Lino, C. A., Harper, J. C., Carney, J. P., & Timlin, J. A. (2018) · Drug Delivery · doi:10.1080/10717544.2018.1474964 · Cited by 1,185
Wang, H.-X., Li, M., Lee, C. M., Chakraborty, S., Kim, H.-W., Bao, G., & Leong, K. W. (2017) · Chemical Reviews · doi:10.1021/acs.chemrev.6b00799 · Cited by 560
Exosome/Liposome-like Nanoparticles: New Carriers for CRISPR Genome Editing in Plants
Alghuthaymi, M. A., Ahmad, A., Khan, Z., Khan, S. H., Ahmed, F. K., Faiz, S., Nepovimova, E., Kuča, K., & Abd-Elsalam, K. A. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22147456 · Cited by 83
Metadata
- Authors
- Horii, T., Tamura, D., Morita, S., Kimura, M., & Hatada, I.
- Affiliations
- Laboratory of Genome Science, Biosignal Genome Resource Center, Institute for Molecular and Cellular Regulation, Gunma University, Gunma 371-8512, Japan
- Venue
- International Journal of Molecular Sciences
- Publisher
- MDPI AG
- Published
- 2013-09-30
- Volume
- 14
- Issue
- 10
- Pages
- 19774-19781
- ISSN
- 1422-0067
- PMC
- PMC3821585
- Cited by
- 90
- References
- 4
- Language
- en
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