ArticleMolecular therapy. Nucleic acids2025
Prime editing corrects the dilated cardiomyopathy causing RBM20-P633L-mutation in human cardiomyocytes.
Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.
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Who cites it
5 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Review
- RBM20 isoform regulation by independent transcription start sites adapts alternative splicing in development and disease.Nature communications · 2026Article
- Epigenetics, Modifiers, and Molecular Noise: Rethinking Pathophysiology in Dilated and Hypertrophic Cardiomyopathies.International journal of molecular sciences · 2026Review
- Treating RBM20-related cardiomyopathy-Ready for prime time?Molecular therapy. Nucleic acids · 2025Article
Corrections and comments
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Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Prime editing (PE) is an innovative next-generation gene editing tool that has therapeutic potential in post-mitotic organs, such as the human heart. However, its applicability and efficiency in non-proliferating cells, e.g., human cardiomyocytes, is not yet established. Here, we apply PE directly in cardiomyocytes differentiated from human induced pluripotent stem cells (hi-CMs) carrying dilated-cardiomyopathy-causing mutations. A target array (TA) containing the mutations LMNA
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Registered trials
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