Targeted gene therapy for the treatment of cardiac dysfunction.

Congestive heart failure (CHF), one of the leading cardiovascular disorders in developed countries, remains a significant therapeutic challenge. Efficacious therapies are few, and the incidence of CHF and associated death rates continue to rise. An interest in the novel therapeutic approach of gene therapy for the treatment of CHF has emerged. Essential elements of successful gene therapy include an appropriate vector for delivering and expressing the gene within the target cell, an optimal protocol for delivery of the gene, and the identification of relevant pathways and molecular targets. Interest in gene therapy for CHF has been directed towards improving cardiomyocyte function through optimization of calcium homeostasis and beta-adrenoreceptor function, and preclinical studies have shown encouraging results. This review will discuss the vectors and mechanisms of gene delivery as well as potential molecular targets for the treatment of CHF. Copyright 2002, Elsevier Science (USA). All rights reserved.