Abstract
Muscular dystrophies are broadly classed as skeletal muscle disease entities of genetic origin. Accordingly, the development and application of gene therapy treatment modalities has focused on skeletal muscle gene replacement. Irrespective of this generalization, most forms of dystrophy are accompanied by progressive cardiomyopathy and cardiac involvement in muscular dystrophies is now recognized as an independent risk for patient morbidity. In this review, we summarize the available murine strains most suitable for modeling the dystrophic myocardium and discuss the use of adenoviral based vector systems as the preferred gene delivery vehicle for modulating dystrophic cardiomyopathy.
Keywords: dystrophy, dystrophic cardiomyopathy, heart, adenovirus, coxsackie virus
Current Gene Therapy
Title: Getting to the Heart of the Matter: Exploring Opportunities for Gene Therapy Treatment of Dystrophic Cardiomyopathy
Volume: 4 Issue: 2
Author(s): Steven M. Kolodziejczyk and Lynn A. Megeney
Affiliation:
Keywords: dystrophy, dystrophic cardiomyopathy, heart, adenovirus, coxsackie virus
Abstract: Muscular dystrophies are broadly classed as skeletal muscle disease entities of genetic origin. Accordingly, the development and application of gene therapy treatment modalities has focused on skeletal muscle gene replacement. Irrespective of this generalization, most forms of dystrophy are accompanied by progressive cardiomyopathy and cardiac involvement in muscular dystrophies is now recognized as an independent risk for patient morbidity. In this review, we summarize the available murine strains most suitable for modeling the dystrophic myocardium and discuss the use of adenoviral based vector systems as the preferred gene delivery vehicle for modulating dystrophic cardiomyopathy.
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Kolodziejczyk M. Steven and Megeney A. Lynn, Getting to the Heart of the Matter: Exploring Opportunities for Gene Therapy Treatment of Dystrophic Cardiomyopathy, Current Gene Therapy 2004; 4 (2) . https://dx.doi.org/10.2174/1566523043346516
DOI https://dx.doi.org/10.2174/1566523043346516 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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