The DOI matches its Crossref record; 7 of the 7 references it cites were found in a registry or an index.
Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease–mediated safe harbor targeting
Open accessAbstract
We have developed induced pluripotent stem cells (iPSCs) from a patient with X-linked chronic granulomatous disease (X-CGD), a defect of neutrophil microbicidal reactive oxygen species (ROS) generation resulting from gp91phox deficiency. We demonstrated that mature neutrophils differentiated from X-CGD iPSCs lack ROS production, reproducing the pathognomonic CGD cellular phenotype. Targeted gene transfer into iPSCs, with subsequent selection and full characterization to ensure no off-target changes, holds promise for correction of monogenic diseases without the insertional mutagenesis caused by multisite integration of viral or plasmid vectors. Zinc finger nuclease–mediated gene targeting of a single-copy gp91phox therapeutic minigene into one allele of the “safe harbor” AAVS1 locus in X-CGD iPSCs without off-target inserts resulted in sustained expression of gp91phox and substantially restored neutrophil ROS production. Our findings demonstrate how precise gene targeting may be applied to correction of X-CGD using zinc finger nuclease and patient iPSCs.
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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
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Self-citation
Cited by
A guide to genome engineering with programmable nucleases
Kim, H., & Kim, J.-S. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3686 · Cited by 1,163
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
Targeted genome editing in human repopulating haematopoietic stem cells
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Safe harbours for the integration of new DNA in the human genome
Sadelain, M., Papapetrou, E. P., & Bushman, F. D. (2012) · Nature Reviews Cancer · doi:10.1038/nrc3179 · Cited by 441
Metadata
- Authors
- Zou, J., Sweeney, C. L., Chou, B.-K., Choi, U., Pan, J., Wang, H., Dowey, S. N., Cheng, L., & Malech, H. L.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2011-05-26
- Volume
- 117
- Issue
- 21
- Pages
- 5561-5572
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2023456870
- Cited by
- 236
- References
- 7
- FWCI
- 14.22
- Type
- journal article
- Language
- en
- Open access
- green
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- CoChat. (2026). Citation check: Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease–mediated safe harbor targeting. https://app.cochat.ai/papers/10.1182/blood-2010-12-328161
- The paper
- Zou, J., Sweeney, C. L., Chou, B.-K., Choi, U., Pan, J., Wang, H., Dowey, S. N., Cheng, L., & Malech, H. L. (2011). Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease–mediated safe harbor targeting. Blood. https://doi.org/10.1182/blood-2010-12-328161
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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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