ArticleNucleic acids research2013
Optimization of scarless human stem cell genome editing.
Article in Nucleic acids research, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 233 papers, 1 of them a synthesis that pooled it.
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Who cites it
233 citing papers in PubMed, 1 synthesis or guideline pooled it.
- 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
- Cyanine-modified ssODNs enhance CRISPR-Cas9 HDR in stem cell embryo models via chromatin and chemical modulation.Nature communications · 2026Article
- Precise CRISPR-Mediated Editing of theInternational journal of molecular sciences · 2026Article
- CTCF couples long-range loop extrusion and diffusion to mediate a diverse Igκ repertoire.Nature communications · 2025Article
- Orthogonal CRISPR systems for targeted integration and multiplex base editing enable nonviral engineering of allogeneic CAR-T cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Optimization of gene knockout approaches and sgRNA selection in hPSCs with inducible Cas9 expression.Scientific reports · 2025Article
- CRISPR-Cas technologies: Emerging tools from research to clinical application.Journal of microbiology (Seoul, Korea) · 2025Review
- Review
- High throughput variant libraries and machine learning yield design rules for retron gene editors.Nucleic acids research · 2025Article
- A protocol for karyotyping and genetic editing of induced pluripotent stem cells with homology-directed repair mediated CRISPR/Cas9.Biology methods & protocols · 2025Article
- High Throughput Variant Libraries and Machine Learning Yield Design Rules for Retron Gene Editors.bioRxiv : the preprint server for biology · 2024Article
- Arp2/3 mediated dynamic lamellipodia of the hPSC colony edges promote liposome-based DNA delivery.Stem cells (Dayton, Ohio) · 2024Article
- Decoding the complexity of on-target integration: characterizing DNA insertions at the CRISPR-Cas9 targeted locus using nanopore sequencing.BMC genomics · 2024Article
- CRISPR-Cas9-generated PTCHD1 2489T>G stem cells recapitulate patient phenotype when undergoing neural induction.HGG advances · 2024Article
- Making gene editing accessible in resource limited environments: recommendations to guide a first-time user.Frontiers in genome editing · 2024Review
- A Functional Schizophrenia-associated genetic variant near thebioRxiv : the preprint server for biology · 2023Article
- CRISPR-Cas9 Methods and Key Considerations in the Production ofCold Spring Harbor protocols · 2023Article
- A Cell-Based Optimised Approach for Rapid and Efficient Gene Editing of Human Pluripotent Stem Cells.International journal of molecular sciences · 2023Article
- Review: Recent Applications of Gene Editing in Fish Species and Aquatic Medicine.Animals : an open access journal from MDPI · 2023Review
- Strategies for generation of mice via CRISPR/HDR-mediated knock-in.Molecular biology reports · 2023Review
173 more citing papers are in PubMed but not listed here.
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Authors and funding
13 authors.
Funding
Abstract
Efficient strategies for precise genome editing in human-induced pluripotent cells (hiPSCs) will enable sophisticated genome engineering for research and clinical purposes. The development of programmable sequence-specific nucleases such as Transcription Activator-Like Effectors Nucleases (TALENs) and Cas9-gRNA allows genetic modifications to be made more efficiently at targeted sites of interest. However, many opportunities remain to optimize these tools and to enlarge their spheres of application. We present several improvements: First, we developed functional re-coded TALEs (reTALEs), which not only enable simple one-pot TALE synthesis but also allow TALE-based applications to be performed using lentiviral vectors. We then compared genome-editing efficiencies in hiPSCs mediated by 15 pairs of reTALENs and Cas9-gRNA targeting CCR5 and optimized ssODN design in conjunction with both methods for introducing specific mutations. We found Cas9-gRNA achieved 7-8× higher non-homologous end joining efficiencies (3%) than reTALENs (0.4%) and moderately superior homology-directed repair efficiencies (1.0 versus 0.6%) when combined with ssODN donors in hiPSCs. Using the optimal design, we demonstrated a streamlined process to generated seamlessly genome corrected hiPSCs within 3 weeks.
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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.