Gene deficient and transgenic animal models of dilated cardiomyopathy elucidate genetic disease mechanisms and demonstrate the potential of novel therapies such as in vivo somatic gene transfer.
This review highlights the utility of gene-deficient and transgenic animal models in understanding genetic dilated cardiomyopathy and exploring novel gene therapies.
Genetic forms of human dilated cardiomyopathy (DCM) are briefly discussed, and a variety of animal models of genetic DCM are presented, some of which are caused by the gene mutations that also cause DCM in humans. The forms of DCM related to mutations or deletion of genes coding for extrasarcomeric or intrasarcomeric proteins, as well as to overexpression or knockout of genes in the beta-adrenergic signaling pathway, are included. Finally, novel approaches to treatment in experimental animal models are discussed, including double transgenesis and newer recombination methods, as well as in vivo somatic gene transfer which, based on initial experiments in animals, seems likely to find eventual application in human cardiac failure.
John Ross (Tue,) conducted a review in Dilated Cardiomyopathy. Gene Deficient and Transgenic Animal Models was evaluated. Gene deficient and transgenic animal models of dilated cardiomyopathy elucidate genetic disease mechanisms and demonstrate the potential of novel therapies such as in vivo somatic gene transfer.
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