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The article investigates transgene-induced cardiotoxicity resulting from high-dose AAV gene transfer, particularly in the context of muscular dystrophies. It highlights that systemic administration of high AAV vector doses can lead to cardiac complications, including myocarditis and even death in some patients. The study emphasizes the need for careful regulation of transgene expression in the heart to mitigate these risks and suggests strategies to prevent cardiotoxicity.
- Author
- Duc Thien Nguyen
- Language
- EN