The DOI matches its Crossref record; 1 of the 1 references it cites were found in a registry or an index.
Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells
Open accessAbstract
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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Cites
Identification of the Cystic Fibrosis Gene: Genetic Analysis
Kerem, B.-S., Rommens, J. M., Buchanan, J. A., Markiewicz, D., Cox, T. K., Chakravarti, A., Buchwald, M., & Tsui, L.-C. (1989) · Science · doi:10.1126/science.2570460 · Cited by 4,142
Cited by
Genome-editing Technologies for Gene and Cell Therapy
Maeder, M. L., & Gersbach, C. A. (2016) · Molecular Therapy · doi:10.1038/mt.2016.10 · Cited by 719
Induced Pluripotent Stem Cells Meet Genome Editing
Hockemeyer, D., & Jaenisch, R. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2016.04.013 · Cited by 499
“…of patient-specific iPSCs followed by in vitro differentiation was also used to generate an isogenic disease model for cystic fibrosis by correcting disease-relevant mutations in CFTR followed by differentiation into airway epithelium (Crane et al., 2015; Firth et al., 2015; Suzuki et al., 2016).”
MethodProgress in therapies for cystic fibrosis
De Boeck, K., & Amaral, M. D. (2016) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(16)00023-0 · Cited by 451
McCauley, K. B., Hawkins, F., Serra, M., Thomas, D. C., Jacob, A., & Kotton, D. N. (2017) · Cell Stem Cell · doi:10.1016/j.stem.2017.03.001 · Cited by 432
Self-citationThe Cellular and Physiological Basis for Lung Repair and Regeneration: Past, Present, and Future
Basil, M. C., Katzen, J., Engler, A. E., Guo, M., Herriges, M. J., Kathiriya, J. J., Windmueller, R., Ysasi, A. B., Zacharias, W. J., Chapman, H. A., et al. (2020) · Cell Stem Cell · doi:10.1016/j.stem.2020.03.009 · Cited by 414
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Metadata
- Authors
- Crane, A. M., Kramer, P., Bui, J. H., Chung, W. J., Li, X. S., Gonzalez-Garay, M. L., Hawkins, F., Liao, W., Mora, D., Choi, S., Wang, J., Sun, H. C., Paschon, D. E., Guschin, D. Y., Gregory, P. D., Kotton, D. N., Holmes, M. C., Sorscher, E. J., & Davis, B. R.
- Venue
- Stem Cell Reports
- Publisher
- Elsevier BV
- Published
- 2015-04
- Volume
- 4
- Issue
- 4
- Pages
- 569-577
- ISSN
- 2213-6711 (ISSN-L)
- OpenAlex
- W2007914388
- Cited by
- 193
- References
- 1
- FWCI
- 9.13
- Type
- journal article
- Language
- en
- Open access
- gold
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- Crane, A. M., Kramer, P., Bui, J. H., Chung, W. J., Li, X. S., Gonzalez-Garay, M. L., Hawkins, F., Liao, W., Mora, D., Choi, S., Wang, J., Sun, H. C., Paschon, D. E., Guschin, D. Y., Gregory, P. D., Kotton, D. N., Holmes, M. C., Sorscher, E. J., & Davis, B. R. (2015). Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells. Stem Cell Reports. https://doi.org/10.1016/j.stemcr.2015.02.005
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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