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**Chapter 8**

**Provisional chapter**

**Gene Therapy for Cardiomyopathies**

**Gene Therapy for Cardiomyopathies**

DOI: 10.5772/intechopen.80478

Heart disease remains the prevalent cause of premature death and accounts for a significant proportion of all hospital admissions. Molecular genetics was integrated quite late in cardiology, but introduced new concepts like sarcolemmopathies, cytoskeletalopathies, and channelopathies useful to better understand the pathophysiology of the development of inherited cardiomyopathies (CMs). As our understanding of the cellular and molecular processes involved in the development and progression of heart disease improved, new therapeutic targets were identified, as were novel approaches such as delivery of genes to replace defective or deficient components and thereby restore structure or function in a diseased heart. We discuss gene addition strategies in the context of monogenic disorders. Moreover, a broader nucleic acid-based modulation of cardiac gene expression for the treatment of cardiac diseases might have larger clinical indications. Inadequate gene delivery remains a potential cause of negative trials. However, progress in innovative formulations and clinically relevant ways of administration should lead to significant progress in the future. Cardiac gene therapy will be integrated

into the therapeutic armamentarium for CM and heart failure.

structure, cardiac function, viral vector, nanoparticles, polymers

**Keywords:** cardiomyopathy, heart failure, genetic disease, gene therapy, cardiac

Cardiomyopathies (CMs) refer basically to diseases of the heart muscle, which can be acquired or inherited [1]. CMs can affect people of all ages. However, people in certain age groups are more likely to have certain types of CMs, as inherited forms predominate in younger indi-

> © 2016 The Author(s). Licensee InTech. This chapter is distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

© 2019 The Author(s). Licensee IntechOpen. This chapter is distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0), which permits unrestricted use,

distribution, and reproduction in any medium, provided the original work is properly cited.

Additional information is available at the end of the chapter

Additional information is available at the end of the chapter

Yves Fromes and Caroline Roques

Yves Fromes and Caroline Roques

http://dx.doi.org/10.5772/intechopen.80478

**1. Introduction: cardiomyopathies**

viduals and acquired diseases increase with age [2–8].

**Abstract**

#### **Gene Therapy for Cardiomyopathies Gene Therapy for Cardiomyopathies**

Yves Fromes and Caroline Roques Yves Fromes and Caroline Roques

Additional information is available at the end of the chapter Additional information is available at the end of the chapter

http://dx.doi.org/10.5772/intechopen.80478

#### **Abstract**

Heart disease remains the prevalent cause of premature death and accounts for a significant proportion of all hospital admissions. Molecular genetics was integrated quite late in cardiology, but introduced new concepts like sarcolemmopathies, cytoskeletalopathies, and channelopathies useful to better understand the pathophysiology of the development of inherited cardiomyopathies (CMs). As our understanding of the cellular and molecular processes involved in the development and progression of heart disease improved, new therapeutic targets were identified, as were novel approaches such as delivery of genes to replace defective or deficient components and thereby restore structure or function in a diseased heart. We discuss gene addition strategies in the context of monogenic disorders. Moreover, a broader nucleic acid-based modulation of cardiac gene expression for the treatment of cardiac diseases might have larger clinical indications. Inadequate gene delivery remains a potential cause of negative trials. However, progress in innovative formulations and clinically relevant ways of administration should lead to significant progress in the future. Cardiac gene therapy will be integrated into the therapeutic armamentarium for CM and heart failure.

DOI: 10.5772/intechopen.80478

**Keywords:** cardiomyopathy, heart failure, genetic disease, gene therapy, cardiac structure, cardiac function, viral vector, nanoparticles, polymers
