ArticleStem cell research2021
A stress-free strategy to correct point mutations in patient iPS cells.
Article in Stem cell research, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed, 7 citations in OpenAlex.
- Generation of a Patient-Derived Inducible Pluripotent Stem Cell Model for Studying theInternational journal of stem cells · 2026Article
- Advances in Research onGenes · 2026Review
- CRISPR Base Editing to Create Potential Charcot-Marie-Tooth Disease Models with High Editing Efficiency: Human Induced Pluripotent Stem Cell HarboringBiomedicines · 2024Article
- CRISPR Del/Rei: a simple, flexible, and efficient pipeline for scarless genome editing.Scientific reports · 2022Article
- On the Road from Phenotypic Plasticity to Stem Cell Therapy.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2021Article
- From in vivo models to in vitro bioengineered neuromuscular junctions for the study of Charcot-Marie-Tooth disease.Journal of tissue engineeringReview
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
When studying patient specific induced pluripotent stem cells (iPS cells) as a disease model, the ideal control is an isogenic line that has corrected the point mutation, instead of iPS cells from siblings or other healthy subjects. However, repairing a point mutation in iPS cells even with the newly developed CRISPR-Cas9 technique remains difficult and time-consuming. Here we report a strategy that makes the Cas9 "knock-in" methodology both hassle-free and error-free. Instead of selecting a Cas9 recognition site close to the point mutation, we chose a site located in the nearest intron. We constructed a donor template with the fragment containing the corrected point mutation as one of the homologous recombination arms flanking a PGK-Puro
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Registered trials
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