Current Achievements in Gene Therapy Strategies and Delivery Systems in Preclinical and Clinical Models of Heart Failure.

purpose of reviewCurrent 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

findingsClinical 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.