ArticleStem cell reports2014
Efficient designer nuclease-based homologous recombination enables direct PCR screening for footprintless targeted human pluripotent stem cells.
Article in Stem cell reports, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 35 citations in OpenAlex.
- Review
- Targeted biallelic integration of an inducible Caspase 9 suicide gene in iPSCs for safer therapies.Molecular therapy. Methods & clinical development · 2022Article
- Towards Biohybrid Lung: Induced Pluripotent Stem Cell Derived Endothelial Cells as Clinically Relevant Cell Source for Biologization.Micromachines · 2021Article
- Efficient Genetic Safety Switches for Future Application of iPSC-Derived Cell Transplants.Journal of personalized medicine · 2021Article
- Genetic Correction of IL-10RB Deficiency Reconstitutes Anti-Inflammatory Regulation in iPSC-Derived Macrophages.Journal of personalized medicine · 2021Article
- Dual Function of iPSC-Derived Pericyte-Like Cells in Vascularization and Fibrosis-Related Cardiac Tissue Remodeling In Vitro.International journal of molecular sciences · 2020Article
- Differentiation of Human Pluripotent Stem Cells into Functional Endothelial Cells in Scalable Suspension Culture.Stem cell reports · 2018Article
- Editing the genome of hiPSC with CRISPR/Cas9: disease models.Mammalian genome : official journal of the International Mammalian Genome Society · 2017Review
- Ex vivo Generation of Genetically Modified Macrophages from Human Induced Pluripotent Stem Cells.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2017Article
- Therapeutic Application of Pluripotent Stem Cells: Challenges and Risks.Frontiers in medicine · 2017Review
- CRISPR/Cas-Mediated Knockin in Human Pluripotent Stem Cells.Methods in molecular biology (Clifton, N.J.) · 2017Article
- Site-Specific Genome Engineering in Human Pluripotent Stem Cells.International journal of molecular sciences · 2016Review
- Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Use of the HPRT gene to study nuclease-induced DNA double-strand break repair.Human molecular genetics · 2015Article
- Pluripotent stem cells for disease modeling and drug screening: new perspectives for treatment of cystic fibrosis?Molecular and cellular pediatrics · 2015Article
- A CRISPR/Cas-Mediated Selection-free Knockin Strategy in Human Embryonic Stem Cells.Stem cell reports · 2015Article
- Genome editing 101: let's go digital.Nature methods · 2014Article
Corrections and comments
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Authors and funding
11 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Genetic engineering of human induced pluripotent stem cells (hiPSCs) via customized designer nucleases has been shown to be significantly more efficient than conventional gene targeting, but still typically depends on the introduction of additional genetic selection elements. In our study, we demonstrate the efficient nonviral and selection-independent gene targeting in human pluripotent stem cells (hPSCs). Our high efficiencies of up to 1.6% of gene-targeted hiPSCs, accompanied by a low background of randomly inserted transgenes, eliminated the need for antibiotic or fluorescence-activated cell sorting selection, and allowed the use of short donor oligonucleotides for footprintless gene editing. Gene-targeted hiPSC clones were established simply by direct PCR screening. This optimized approach allows targeted transgene integration into safe harbor sites for more predictable and robust expression and enables the straightforward generation of disease-corrected, patient-derived iPSC lines for research purposes and, ultimately, for future clinical applications.
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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.