ArticleFrontiers in cell and developmental biology2020
Base Editing Mediated Generation of Point Mutations Into Human Pluripotent Stem Cells for Modeling Disease.
Article in Frontiers in cell and developmental biology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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Who cites it
21 citing papers in PubMed.
- CRISPR/Cas Systems: Biological Basis and Genome Editing Applications.Methods in molecular biology (Clifton, N.J.) · 2027Review
- Induced pluripotent stem cells from discovery to translation.Nature medicine · 2026Review
- Search-and-remove genome editing allows selection of cells by DNA sequence.Nature communications · 2025Article
- The 2SP Site Mutation in the Bovine Natural Resistance-Associated Macrophage 1 Promoter Exhibits Antituberculosis Potential.International journal of molecular sciences · 2025Article
- Enhanced genome editing with a Streptococcus equinus Cas9.Communications biology · 2025Article
- Generation of an Isogenic Hereditary Hemorrhagic Telangiectasia Model via Prime Editing in Human Induced Pluripotent Stem Cells.International journal of stem cells · 2024Article
- Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors.Molecular therapy. Nucleic acids · 2024Article
- Enhancing genome editing in hPSCs through dual inhibition of DNA damage response and repair pathways.Nature communications · 2024Article
- Phage-assisted evolution of compact Cas9 variants targeting a simple NNG PAM.Nature chemical biology · 2024Article
- Recent Research Trends in Stem Cells Using CRISPR/Cas-Based Genome Editing Methods.International journal of stem cells · 2024Review
- Efficient gene editing in induced pluripotent stem cells enabled by an inducible adenine base editor with tunable expression.Scientific reports · 2023Article
- Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells.Stem cell reviews and reports · 2023Review
- Gene editing with 'pencil' rather than 'scissors' in human pluripotent stem cells.Stem cell research & therapy · 2023Review
- Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders.International journal of molecular sciences · 2022Review
- Identification of SaCas9 orthologs containing a conserved serine residue that determines simple NNGG PAM recognition.PLoS biology · 2022Article
- Article
- KCNQ1-deficient and KCNQ1-mutant human embryonic stem cell-derived cardiomyocytes for modeling QT prolongation.Stem cell research & therapy · 2022Article
- Compact SchCas9 Recognizes the Simple NNGR PAM.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2022Article
- How Functional Genomics Can Keep Pace With VUS Identification.Frontiers in cardiovascular medicine · 2022Review
- Gene Therapy: The Next-Generation Therapeutics and Their Delivery Approaches for Neurological Disorders.Frontiers in genome editing · 2022Review
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9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Human pluripotent stem cells (hPSCs) are a powerful platform for disease modeling and drug discovery. However, the introduction of known pathogenic mutations into hPSCs is a time-consuming and labor-intensive process. Base editing is a newly developed technology that enables facile introduction of point mutations into specific loci within the genome of living cells. Here, we design an all-in-one episomal vector that expresses a single guide RNA (sgRNA) with an adenine base editor (ABE) or a cytosine base editor (CBE). Both ABE and CBE can efficiently introduce mutations into cells, A-to-G and C-to-T, respectively. We introduce disease-specific mutations of long QT syndrome into hPSCs to model LQT1, LQT2, and LQT3. Electrophysiological analysis of hPSC-derived cardiomyocytes (hPSC-CMs) using multi-electrode arrays (MEAs) reveals that edited hPSC-CMs display significant increases in duration of the action potential. Finally, we introduce the novel Brugada syndrome-associated mutation into hPSCs, demonstrating that this mutation can cause abnormal electrophysiology. Our study demonstrates that episomal encoded base editors (epi-BEs) can efficiently generate mutation-specific disease hPSC models.
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