Curr Heart Fail Rep. 2025 Nov 29;22(1):43. doi: 10.1007/s11897-025-00734-9.
ABSTRACT
PURPOSE OF REVIEW: Current treatment strategies for heart failure primarily aim to slow disease progression. In the case of rare genetic disorders with associated cardiomyopathies, management is largely supportive. Gene therapy offers a promising alternative that is potentially curative. This review explores gene therapy strategies, vector selection and relevant molecular targets. We also examine pre-clinical and clinical trials investigating gene therapy in cardiomyopathies.
RECENT FINDINGS: Clinical trials for several genetic disorders-including Danon disease, Fabry disease and Duchenne muscular dystrophy-have demonstrated encouraging results. However, with the use of viral vectors and associated immunogenicity eliciting a strong inflammatory response in some patients, there remains a substantial risk of morbidity and mortality. For rare diseases, gene therapy represents a potentially curative, one-time treatment strategy. As the field advances, further work is required to improve vector technology, reduce immunogenicity, decrease off-target effects and address the associated ethical considerations.
PMID:41317268 | DOI:10.1007/s11897-025-00734-9