Gene therapy: targeting the myocardium

• Published in: Heart (British Cardiac Society) Vol. 94; no. 1; pp. 89 - 99
• Main Authors: Lyon, A R, Sato, M, Hajjar, R J, Samulski, R J, Harding, S E
• Format: Journal Article
• Language: English
• Published: London BMJ Publishing Group Ltd and British Cardiovascular Society 01.01.2008; BMJ; BMJ Publishing Group LTD
• Subjects: adeno-associated virus; adenovirus; Artificial chromosomes; Biological and medical sciences; Cardiology. Vascular system; Cardiomyocytes; Cardiomyopathies - therapy; Deoxyribonucleic acid; Dependovirus - genetics; Design; Disease; DNA; Efficiency; Gene expression; Gene Expression Regulation, Viral; Gene therapy; Gene Transfer Techniques; Genetic Therapy - methods; Genetic Vectors; Heart; Humans; Lentivirus - genetics; Medical sciences; Mortality; myocardium; Practice Guidelines as Topic; Proteins; Retroviridae - genetics; Vectors (Biology); Viruses; Treatment; Targeting; Myocardium; Circulatory system; Cardiology; Gene therapy; Phlebology
• ISSN: 1355-6037; 1468-201X
• DOI: 10.1136/hrt.2007.116483

Effective clinical delivery of gene therapy to the heart requires understanding and design of complex biological systems to deliver therapeutic gene expression. The development of vectors that specifically target the myocardium, in particular bioengineered recombinant viruses, has improved the efficiency of gene delivery to the heart. These tools, coupled with advances in selection and design of the genetic payload, have led to effective cardiac gene therapy in preclinical models. This technology is currently translating to the clinic with a new wave of gene therapy trials for myocardial disease.