ArticleMethods in enzymology2014
Genome editing in human stem cells.
Article in Methods in enzymology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 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.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
52 citing papers in PubMed, 92 citations in OpenAlex.
- CRISPR/Cas9-based repair of a heterozygous HNF1A mutation in patient-derived hiPSCs.Human genetics · 2026Article
- Plasmid2MC: efficient cell-free generation of high-purity minicircle DNA for genome editing in mammalian cells.Communications biology · 2025Article
- Integration of xeno-free single-cell cloning in CRISPR-mediated DNA editing of human iPSCs improves homogeneity and methodological efficiency of cellular disease modeling.Stem cell reports · 2023Article
- Article
- "Off-the-Shelf" Allogeneic CAR Cell Therapy-Neglected HvG Effect.Current treatment options in oncology · 2023Review
- Fluorescent hiPSC-derived MYH6-mScarlet cardiomyocytes for real-time tracking, imaging, and cardiotoxicity assays.Cell biology and toxicology · 2023Article
- Homozygous might be hemizygous: CRISPR/Cas9 editing in iPSCs results in detrimental on-target defects that escape standard quality controls.Stem cell reports · 2022Article
- A framework for developing sex-specific engineered heart models.Nature reviews. Materials · 2022Review
- Strategies to Improve the Safety of iPSC-Derived β Cells for β Cell Replacement in Diabetes.Transplant international : official journal of the European Society for Organ Transplantation · 2022Review
- Microfluidic processing of stem cells for autologous cell replacement.Stem cells translational medicine · 2021Review
- Targeted mutagenesis in human iPSCs using CRISPR genome-editing tools.Methods (San Diego, Calif.) · 2021Article
- Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair proteins.Nature communications · 2021Article
- Correction of Hemoglobin E/Beta-Thalassemia Patient-Derived iPSCs Using CRISPR/Cas9.Methods in molecular biology (Clifton, N.J.) · 2021Article
- Genetically Modified Mesenchymal Stromal/Stem Cells: Application in Critical Illness.Stem cell reviews and reports · 2020Review
- Modeling Psychiatric Disorder Biology with Stem Cells.Current psychiatry reports · 2020Review
- Generation of New Isogenic Models of Huntington's Disease Using CRISPR-Cas9 Technology.International journal of molecular sciences · 2020Article
- Genomic Engineering in Human Hematopoietic Stem Cells: Hype or Hope?Frontiers in genome editing · 2020Review
- Genetic predispositions of Parkinson's disease revealed in patient-derived brain cells.NPJ Parkinson's disease · 2020Review
- Brain Organoids as Tools for Modeling Human Neurodevelopmental Disorders.Physiology (Bethesda, Md.) · 2019Review
- Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS.Diseases (Basel, Switzerland) · 2019Review
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
The use of custom-engineered sequence-specific nucleases (including CRISPR/Cas9, ZFN, and TALEN) allows genetic changes in human cells to be easily made with much greater efficiency and precision than before. Engineered double-stranded DNA breaks can efficiently disrupt genes, or, with the right donor vector, engineer point mutations and gene insertions. However, a number of design considerations should be taken into account to ensure maximum gene targeting efficiency and specificity. This is especially true when engineering human embryonic stem or induced pluripotent stem cells (iPSCs), which are more difficult to transfect and less resilient to DNA damage than immortalized tumor cell lines. Here, we describe a protocol for easily engineering genetic changes in human iPSCs, through which we typically achieve targeting efficiencies between 1% and 10% without any subsequent selection steps. Since this protocol only uses the simple transient transfection of plasmids and/or single-stranded oligonucleotides, most labs will easily be able to perform it. We also describe strategies for identifying, cloning, and genotyping successfully edited cells, and how to design the optimal sgRNA target sites and donor vectors. Finally, we discuss alternative methods for gene editing including viral delivery vectors, Cas9 nickases, and orthogonal Cas9 systems.
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What OpenQuestion holds
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.