The DOI matches its Crossref record; 16 of the 16 references it cites were found in a registry or an index.
An iCRISPR Platform for Rapid, Multiplexable, and Inducible Genome Editing in Human Pluripotent Stem Cells
Open accessAbstract
Summary Human pluripotent stem cells (hPSCs) offer a unique platform for elucidating the genes and molecular pathways that underlie complex traits and diseases. To realize this promise, methods for rapid and controllable genetic manipulations are urgently needed. By combining two newly developed gene-editing tools, the TALEN and CRISPR/Cas systems, we have developed a novel genome-engineering platform in hPSCs, which we named iCRISPR. iCRISPR enabled rapid and highly efficient generation of biallelic knockout hPSCs for loss-of-function studies, as well as homozygous knockin hPSCs with specific nucleotide alterations for precise modeling of disease conditions. We further demonstrate, for the first time, efficient one-step generation of double- and triple-gene knockout hPSC lines, as well as stage-specific inducible gene knockout during hPSC differentiation. Thus the iCRISPR platform is uniquely suited for dissection of complex genetic interactions and pleiotropic gene functions in human disease studies, and has the potential to support high-throughput genetic analysis in hPSCs.
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Cites
A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
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Meta-Analysis of 74,046 Individuals Identifies 11 New Susceptibility Loci for Alzheimer’s Disease
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“, nCas9, dCas9, or dCas9-KRAB) (Gilbert et al., 2013; Mali et al., 2013a; Qi et al., 2013; Ran et al., 2013b), one may repurpose iCRISPR for additional tasks such as gene regulation.”
BackgroundWang, H., Yang, H., Shivalila, C. S., Dawlaty, M. M., Cheng, A. W., Zhang, F., & Jaenisch, R. (2013) · Cell · doi:10.1016/j.cell.2013.04.025 · Cited by 3,495
High-Frequency off-Target Mutagenesis Induced by CRISPR-Cas Nucleases in Human Cells
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“Recent studies suggest that the CRISPR/Cas system may have offtarget effects (Cho et al., 2014; Fu et al., 2013; Hsu et al., 2013; Mali et al., 2013a; Pattanayak et al., 2013), potentially confounding genetic studies in hPSCs.”
BackgroundEfficient genome editing in zebrafish using a CRISPR-Cas system
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“We were able to differentiate iCas9 hESCs first into definitive endoderm (DE) and subsequently into pancreatic progenitor (PP) and insulin-expressing b-like cells based on an established protocol (Kroon et al., 2008) (Figures S5A and S5B).”
MethodMali, P., Aach, J., Stranges, P. B., Esvelt, K. M., Moosburner, M., Kosuri, S., Yang, L., & Church, G. M. (2013) · Nature Biotechnology · doi:10.1038/nbt.2675 · Cited by 1,752
TALENs: A widely applicable technology for targeted genome editing
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“Recent studies suggest that the CRISPR/Cas system may have offtarget effects (Cho et al., 2014; Fu et al., 2013; Hsu et al., 2013; Mali et al., 2013a; Pattanayak et al., 2013), potentially confounding genetic studies in hPSCs.”
Background
Cited by
The next generation of CRISPR–Cas technologies and applications
Pickar-Oliver, A., & Gersbach, C. A. (2019) · Nature Reviews Molecular Cell Biology · doi:10.1038/s41580-019-0131-5 · Cited by 1,459
Chu, V. T., Weber, T., Wefers, B., Wurst, W., Sander, S., Rajewsky, K., & Kühn, R. (2015) · Nature Biotechnology · doi:10.1038/nbt.3198 · Cited by 1,210
CRISPR/Cas9 in Genome Editing and Beyond
Wang, H., La Russa, M., & Qi, L. S. (2016) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-060815-014607 · Cited by 1,110
p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells
Ihry, R. J., Worringer, K. A., Salick, M. R., Frias, E., Ho, D., Theriault, K., Kommineni, S., Chen, J., Sondey, M., Ye, C., et al. (2018) · Nature Medicine · doi:10.1038/s41591-018-0050-6 · Cited by 978
CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs
Mandegar, M. A., Huebsch, N., Frolov, E. B., Shin, E., Truong, A., Olvera, M. P., Chan, A. H., Miyaoka, Y., Holmes, K., Spencer, C. I., et al. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2016.01.022 · Cited by 525
“However, CRISPRn may not be the most robust system for loss-of-function studies, because it is limited by the number of cells within a population that do not produce knockoutphenotypes (González et al., 2014).”
Metadata
- Authors
- González, F., Zhu, Z., Shi, Z.-D., Lelli, K., Verma, N., Li, Q. V., & Huangfu, D.
- Venue
- Cell Stem Cell
- Publisher
- Elsevier BV
- Published
- 2014-08
- Volume
- 15
- Issue
- 2
- Pages
- 215-226
- ISSN
- 1875-9777 (ISSN-L)
- 1934-5909
- OpenAlex
- W2114232807
- Cited by
- 503
- References
- 16
- FWCI
- 19.35
- Type
- journal article
- Language
- en
- Open access
- bronze
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