Gene therapy targeting INa to treat life-threatening arrhythmias: beyond proof-of-concept?
This editorial refers to ‘SCN10A-short gene therapy to restore conduction and protect against malignant cardiac arrhythmias’, by J. Wang et al., https://doi.org/10.1093/eurheartj/ehaf053.Ventricular arrhythmias are a significant cause of morbidity and mortality in patients with impaired cardiac func...
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
7 May 2025
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| In: |
European heart journal
Year: 2025, Volume: 46, Issue: 18, Pages: 1763-1765 |
| ISSN: | 1522-9645 |
| DOI: | 10.1093/eurheartj/ehae930 |
| Online Access: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1093/eurheartj/ehae930
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| Author Notes: | Patrick Lugenbiel |
| Summary: | This editorial refers to ‘SCN10A-short gene therapy to restore conduction and protect against malignant cardiac arrhythmias’, by J. Wang et al., https://doi.org/10.1093/eurheartj/ehaf053.Ventricular arrhythmias are a significant cause of morbidity and mortality in patients with impaired cardiac function and structural heart disease, often leading to sudden cardiac death.1 Effective management and drug therapies of cardiac arrhythmias remains a significant challenge in cardiovascular medicine. Gene therapy has emerged as a promising solution by targeting defective cellular signalling pathways. Researchers hypothesized that, unlike protein-based drugs which may require repeated infusions, gene-based therapies delivered to long-lived cells could enable sustained production of endogenous proteins. Since the first reports of successful gene transfer, in 1972 Friedmann et al. provided a prophetic account of the potential and challenges associated with using gene therapy to treat inherited monogenic disorders.2 In recent years, multiple approaches have been developed to treat both supraventricular and ventricular arrhythmias using gene therapy.3-7 Gene therapy for heart rhythm disorders is currently in pre-clinical evaluation, and no antiarrhythmic gene therapy drugs are commercially available or have been tested in clinical trials yet. |
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| Item Description: | Im Titel ist "Na" bei "INa" tiefgestellt Online veröffentlicht am 21. Februar 2025 Gesehen am 28.07.2025 |
| Physical Description: | Online Resource |
| ISSN: | 1522-9645 |
| DOI: | 10.1093/eurheartj/ehae930 |