ArticleScientific reports2017
Targeted Genome Replacement via Homology-directed Repair in Non-dividing Cardiomyocytes.
Article in Scientific reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.
What it found
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Who cites it
30 citing papers in PubMed, 46 citations in OpenAlex.
- Design and in silico validation of donor DNA for RNA-guided recombinase-mediated knockout of mstnb gene in Labeo rohita.PloS one · 2026Article
- Gene Editing Applications as Future Cardiovascular Therapies.Annual review of genetics · 2025Review
- CRISPR-based gene editing in plants: Focus on reagents and their delivery tools.BioImpacts : BI · 2025Review
- CRISPR-Cas9-mediated homology-directed repair for precise gene editing.Molecular therapy. Nucleic acids · 2024Review
- CRISPR/Cas system-mediated base editing in crops: recent developments and future prospects.Plant cell reports · 2024Review
- Precise genome-editing in human diseases: mechanisms, strategies and applications.Signal transduction and targeted therapy · 2024Review
- Disease modeling of desmosome-related cardiomyopathy using induced pluripotent stem cell-derived cardiomyocytes.World journal of stem cells · 2023Review
- Recent advances in CRISPR-based genome editing technology and its applications in cardiovascular research.Military Medical Research · 2023Review
- Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities.Journal of nanobiotechnology · 2022Review
- Human-Induced Pluripotent Stem Cell-Derived Cardiomyocyte Model forCirculation. Genomic and precision medicine · 2022Article
- The use of new CRISPR tools in cardiovascular research and medicine.Nature reviews. Cardiology · 2022Review
- CRISPR Modeling and Correction of Cardiovascular Disease.Circulation research · 2022Review
- Efficient In Vivo Homology-Directed Repair Within Cardiomyocytes.Circulation · 2022Article
- Modeling reduced contractility and impaired desmosome assembly due to plakophilin-2 deficiency using isogenic iPS cell-derived cardiomyocytes.Stem cell reports · 2022Article
- Identification of an amino-terminus determinant critical for ryanodine receptor/Ca2+ release channel function.Cardiovascular research · 2021Article
- Aspects of Gene Therapy Products Using Current Genome-Editing Technology in Japan.Human gene therapy · 2020Review
- Adeno-associated virus-mediated gene delivery promotes S-phase entry-independent precise targeted integration in cardiomyocytes.Scientific reports · 2020Article
- High rate of HDR in gene editing of p.(Thr158Met) MECP2 mutational hotspot.European journal of human genetics : EJHG · 2020Article
- Promoter Orientation within an AAV-CRISPR Vector Affects Cas9 Expression and Gene Editing Efficiency.The CRISPR journal · 2020Article
- Gene therapy for inherited arrhythmias.Cardiovascular research · 2020Review
Corrections and comments
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Authors and funding
12 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Although high-throughput sequencing can elucidate the genetic basis of hereditary cardiomyopathy, direct interventions targeting pathological mutations have not been established. Furthermore, it remains uncertain whether homology-directed repair (HDR) is effective in non-dividing cardiomyocytes. Here, we demonstrate that HDR-mediated genome editing using CRISPR/Cas9 is effective in non-dividing cardiomyocytes. Transduction of adeno-associated virus (AAV) containing sgRNA and repair template into cardiomyocytes constitutively expressing Cas9 efficiently introduced a fluorescent protein to the C-terminus of Myl2. Imaging-based sequential evaluation of endogenously tagged protein revealed that HDR occurs in cardiomyocytes, independently of DNA synthesis. We sought to repair a pathological mutation in Tnnt2 in cardiomyocytes of cardiomyopathy model mice. An sgRNA that avoided the mutated exon minimized deleterious effects on Tnnt2 expression, and AAV-mediated HDR achieved precise genome correction at a frequency of ~12.5%. Thus, targeted genome replacement via HDR is effective in non-dividing cardiomyocytes, and represents a potential therapeutic tool for targeting intractable cardiomyopathy.
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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.