Key result
AAV-based gene therapy for inherited cardiomyopathies is associated with immunological reactions and potential toxic effects, particularly vector-related myocarditis.
Why the study?
Host immune responses to AAV and gene products pose delivery and safety challenges, including potential toxic effects such as myocarditis.
AAV-based gene therapy for inherited cardiomyopathies poses safety challenges due to host immune responses, notably myocarditis.
Warrants strict immunomodulation protocols in early-phase trials; leaves open the.
Gene therapy is a technique to correct genetic abnormalities, through introduction of a functional gene or through direct genome editing. Adeno-associated virus (AAV)-mediated gene replacement shows promise for targeted therapies in treatment of inherited cardiomyopathies and is the most used approach in clinical trials. However, immune responses from the host to the virus and gene product pose delivery and safety challenges. This review explores the immunological reactions to AAV-based gene therapy, their potential toxic effects, with a focus on myocarditis, and future directions for gene therapy.
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Silver et al. (2023) conducted a review in Inherited cardiomyopathies. AAV-based gene therapy was evaluated. AAV-based gene therapy for inherited cardiomyopathies is associated with immunological reactions and potential toxic effects, particularly vector-related myocarditis.
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