ArticleStem cells and development2013
Generation and genetic engineering of human induced pluripotent stem cells using designed zinc finger nucleases.
Article in Stem cells and development, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
22 citing papers in PubMed, 44 citations in OpenAlex.
- Deciphering reprogramming efficiency in human induced pluripotent stem cells: insights from the generation of 150 cell lines.Frontiers in immunology · 2025Article
- Targeted Gene Insertion for Functional CFTR Restoration in Airway Epithelium.Frontiers in genome editing · 2022Article
- Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF.Pediatric pulmonology · 2021Review
- Correction of Airway Stem Cells: Genome Editing Approaches for the Treatment of Cystic Fibrosis.Human gene therapy · 2020Review
- Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Modeling Cardiac Disease Mechanisms Using Induced Pluripotent Stem Cell-Derived Cardiomyocytes: Progress, Promises and Challenges.International journal of molecular sciences · 2020Review
- Gene and Base Editing as a Therapeutic Option for Cystic Fibrosis-Learning from Other Diseases.Genes · 2019Review
- Recent advances in the use of ZFN-mediated gene editing for human gene therapy.Cell & gene therapy insights · 2017Article
- Impact of gene editing on the study of cystic fibrosis.Human genetics · 2016Review
- Concurrent progress of reprogramming and gene correction to overcome therapeutic limitation of mutant ALK2-iPSC.Experimental & molecular medicine · 2016Article
- Introducing precise genetic modifications into human 3PN embryos by CRISPR/Cas-mediated genome editing.Journal of assisted reproduction and genetics · 2016Article
- Origins of Programmable Nucleases for Genome Engineering.Journal of molecular biology · 2016Review
- Choices for Induction of Pluripotency: Recent Developments in Human Induced Pluripotent Stem Cell Reprogramming Strategies.Stem cell reviews and reports · 2016Review
- Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model.PloS one · 2016Article
- Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations.Stem cells international · 2016Review
- Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium.Nature communications · 2015Article
- Generation of CCR5-defective CD34 cells from ZFN-driven stop codon-integrated mesenchymal stem cell clones.Journal of biomedical science · 2015Article
- Translation: screening for novel therapeutics with disease-relevant cell types derived from human stem cell models.Biological psychiatry · 2014Review
- Use of the heteroduplex mobility assay and cell sorting to select genome sequences of the CCR5 gene in HEK 293T cells edited by transcription activator-like effector nucleases.Genetics and molecular biology · 2014Article
- Zinc finger nuclease-expressing baculoviral vectors mediate targeted genome integration of reprogramming factor genes to facilitate the generation of human induced pluripotent stem cells.Stem cells translational medicine · 2013Article
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 3 countries.
Funding
Abstract
Zinc finger nucleases (ZFNs) have become powerful tools to deliver a targeted double-strand break at a pre-determined chromosomal locus in order to insert an exogenous transgene by homology-directed repair. ZFN-mediated gene targeting was used to generate both single-allele chemokine (C-C motif) receptor 5 (CCR5)-modified human induced pluripotent stem cells (hiPSCs) and biallele CCR5-modified hiPSCs from human lung fibroblasts (IMR90 cells) and human primary cord blood mononuclear cells (CBMNCs) by site-specific insertion of stem cell transcription factor genes flanked by LoxP sites into the endogenous CCR5 locus. The Oct4 and Sox2 reprogramming factors, in combination with valproic acid, induced reprogramming of human lung fibroblasts to form CCR5-modified hiPSCs, while 5 factors, Oct4/Sox2/Klf4/Lin28/Nanog, induced reprogramming of CBMNCs. Subsequent Cre recombinase treatment of the CCR5-modified IMR90 hiPSCs resulted in the removal of the Oct4 and Sox2 transgenes. Further genetic engineering of the single-allele CCR5-modified IMR90 hiPSCs was achieved by site-specific addition of the large CFTR transcription unit to the remaining CCR5 wild-type allele, using CCR5-specific ZFNs and a donor construct containing tdTomato and CFTR transgenes flanked by CCR5 homology arms. CFTR was expressed efficiently from the endogenous CCR5 locus of the CCR5-modified tdTomato/CFTR hiPSCs. These results suggest that it might be feasible to use ZFN-evoked strategies to (1) generate precisely targeted genetically well-defined patient-specific hiPSCs, and (2) then to reshape their function by targeted addition and expression of therapeutic genes from the CCR5 chromosomal locus for autologous cell-based transgene-correction therapy to treat various recessive monogenic human diseases in the future.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.