The DOI matches its Crossref record; 10 of the 10 references it cites were found in a registry or an index.
Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs
Open accessAbstract
Lung disease is a major cause of death in the United States, with current therapeutic approaches serving only to manage symptoms. The most common chronic and life-threatening genetic disease of the lung is cystic fibrosis (CF) caused by mutations in the cystic fibrosis transmembrane regulator (CFTR). We have generated induced pluripotent stem cells (iPSCs) from CF patients carrying a homozygous deletion of F508 in the CFTR gene, which results in defective processing of CFTR to the cell membrane. This mutation was precisely corrected using CRISPR to target corrective sequences to the endogenous CFTR genomic locus, in combination with a completely excisable selection system, which significantly improved the efficiency of this correction. The corrected iPSCs were subsequently differentiated to mature airway epithelial cells where recovery of normal CFTR expression and function was demonstrated. This isogenic iPSC-based model system for CF could be adapted for the development of new therapeutic approaches.
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Cites
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Induced pluripotent stem cell technology: a decade of progress.
Shi, Y., Inoue, H., Wu, J. C., & Yamanaka, S. (2017) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2016.245 · Cited by 1,525
Applications of CRISPR technologies in research and beyond
Barrangou, R., & Doudna, J. A. (2016) · Nature Biotechnology · doi:10.1038/nbt.3659 · Cited by 1,005
Disease modeling in stem cell-derived 3D organoid systems
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Genome-editing Technologies for Gene and Cell Therapy
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Metadata
- Authors
- Firth, A. L., Menon, T., Parker, G. S., Qualls, S. J., Lewis, B. M., Ke, E., Dargitz, C. T., Wright, R., Khanna, A., Gage, F. H., & Verma, I. M.
- Venue
- Cell Reports
- Publisher
- Elsevier BV
- Published
- 2015-09
- Volume
- 12
- Issue
- 9
- Pages
- 1385-1390
- ISSN
- 2211-1247 (ISSN-L)
- OpenAlex
- W1617270423
- Cited by
- 326
- References
- 10
- FWCI
- 24.02
- Type
- journal article
- Language
- en
- Open access
- gold
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