ArticleNature protocols2013
Seamless genome editing in human pluripotent stem cells using custom endonuclease-based gene targeting and the piggyBac transposon.
Article in Nature protocols, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers, 1 of them a synthesis that pooled it.
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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
48 citing papers in PubMed, 1 synthesis or guideline pooled it, 88 citations in OpenAlex.
- Identification and single-base gene-editing functional validation of a cis-EPO variant as a genetic predictor for EPO-increasing therapies.American journal of human genetics · 2022Pooled it
- Single cell transcriptional perturbome in pluripotent stem cell models.Molecular systems biology · 2026Article
- Genetically Engineered Hypoimmune Human Muscle Progenitor Cells Can Reduce Immune Rejection.Cell proliferation · 2025Article
- Article
- Enrichment strategies to enhance genome editing.Journal of biomedical science · 2023Review
- A Cell-Based Optimised Approach for Rapid and Efficient Gene Editing of Human Pluripotent Stem Cells.International journal of molecular sciences · 2023Article
- Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders.International journal of molecular sciences · 2022Review
- CRISPR and iPSCs: Recent Developments and Future Perspectives in Neurodegenerative Disease Modelling, Research, and Therapeutics.Neurotoxicity research · 2022Review
- CRISPR-Cas9-directed gene tagging using a single integrase-defective lentiviral vector carrying a transposase-based Cas9 off switch.Molecular therapy. Nucleic acids · 2022Article
- CRISPR Del/Rei: a simple, flexible, and efficient pipeline for scarless genome editing.Scientific reports · 2022Article
- CRISPR-targeted genome editing of human induced pluripotent stem cell-derived hepatocytes for the treatment of Wilson's disease.JHEP reports : innovation in hepatology · 2022Article
- Extracellular LGALS3BP regulates neural progenitor position and relates to human cortical complexity.Nature communications · 2021Article
- MAPRE2 mutations result in altered human cranial neural crest migration, underlying craniofacial malformations in CSC-KT syndrome.Scientific reports · 2021Article
- CRISPR/Cas System and Factors Affecting Its Precision and Efficiency.Frontiers in cell and developmental biology · 2021Review
- Applications ofStem cells international · 2021Review
- Potential of transposon-mediated cellular reprogramming towards cell-based therapies.World journal of stem cells · 2020Review
- Genome editing technologies to treat rare liver diseases.Translational gastroenterology and hepatology · 2020Review
- A new patient-derived iPSC model for dystroglycanopathies validates a compound that increases glycosylation of α-dystroglycan.EMBO reports · 2019Article
- Chromatin compartment dynamics in a haploinsufficient model of cardiac laminopathy.The Journal of cell biology · 2019Article
- Multiomics Analyses of HNF4α Protein Domain Function during Human Pluripotent Stem Cell Differentiation.iScience · 2019Article
Corrections and comments
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Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
I report here a detailed protocol for seamless genome editing using the piggyBac transposon in human pluripotent stem cells (hPSCs). Recent advances in custom endonucleases have enabled us to routinely perform genome editing in hPSCs. Conventional approaches use the Cre/loxP system that leaves behind residual sequences in the targeted genome. I used the piggyBac transposon to seamlessly remove a drug selection cassette and demonstrated safe genetic correction of a mutation causing α-1 antitrypsin deficiency in patient-derived hPSCs. An alternative approach to using the piggyBac transposon to correct mutations involves using single-stranded oligonucleotides, which is a faster process to complete. However, this experimental procedure is rather complicated and it may be hard to achieve homozygous modifications. In contrast, using the piggyBac transposon with drug selection-based enrichment of genetic modifications, as described here, is simple and can yield multiple correctly targeted clones, including homozygotes. Although two rounds of genetic manipulation are required to achieve homozygote modifications, the entire process takes ∼3 months to complete.
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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.