ArticleNature protocols2016
Modeling and correction of structural variations in patient-derived iPSCs using CRISPR/Cas9.
Article in Nature protocols, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 29 citations in OpenAlex.
- Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCs.Experimental & molecular medicine · 2025Article
- Efficient correction ofMolecular therapy. Nucleic acids · 2023Article
- Insulin resistance in diabetes: The promise of using induced pluripotent stem cell technology.World journal of stem cells · 2021Review
- Reversal of the Inflammatory Responses in Fabry Patient iPSC-Derived Cardiovascular Endothelial Cells by CRISPR/Cas9-Corrected Mutation.International journal of molecular sciences · 2021Article
- Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease.Molecular therapy. Methods & clinical development · 2020Review
- Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders.Molecular therapy. Methods & clinical development · 2020Article
- KCNJ13 Gene Deletion Impairs Cell Alignment and Phagocytosis in Retinal Pigment Epithelium Derived from Human-Induced Pluripotent Stem Cells.Investigative ophthalmology & visual science · 2020Article
- Review
- Restoration of FVIII expression by targeted gene insertion in the FVIII locus in hemophilia A patient-derived iPSCs.Experimental & molecular medicine · 2019Article
- Mechanical influences on cardiovascular differentiation and disease modeling.Experimental cell research · 2019Review
- Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system.Stem cell research & therapy · 2018Article
- Targeted Reactivation ofFrontiers in molecular neuroscience · 2018Article
- CRISPR Editing in Biological and Biomedical Investigation.Journal of cellular biochemistry · 2017Review
- Editing the genome of hiPSC with CRISPR/Cas9: disease models.Mammalian genome : official journal of the International Mammalian Genome Society · 2017Review
- Efficient Generation of Multi-gene Knockout Cell Lines and Patient-derived Xenografts Using Multi-colored Lenti-CRISPR-Cas9.Bio-protocol · 2017Article
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Genome engineering technology using engineered nucleases has been rapidly developing, enabling the efficient correction of simple mutations. However, the precise correction of structural variations (SVs) such as large inversions remains limited. Here we describe a detailed procedure for the modeling or correction of large chromosomal rearrangements and short nucleotide repeat expansions using engineered nucleases in human induced pluripotent stem cells (hiPSCs) from a healthy donor and patients with SVs. This protocol includes the delivery of engineered nucleases with no donor template to hiPSCs, and genotyping and derivation/characterization of gene-manipulated hiPSC clones. With engineered nucleases, genomic inversions, reversions, and deletions of short nucleotide expansions can be identified in 2 weeks, and desired clones can be generated in as little as 3-4 weeks. This protocol enables the correction of large inverted segments and short nucleotide repeat expansions in diseases such as hemophilia A, fragile X syndrome, Hunter syndrome, and Friedreich's ataxia.
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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.