TECHNOLOGY AND GUIDELINES

Gene therapy: targeting the myocardium

A R Lyon^1,2, M Sato¹, R J Hajjar², R J Samulski³, S E Harding¹

¹ Department of Cardiac Medicine, National Heart and Lung Institute, London, UK
² Cardiovascular Research Centre, Mount Sinai Hospital, New York, USA
³ Gene Therapy Centre, University of North Carolina, Chapel Hill, North Carolina, USA

Correspondence to:
Dr Alexander Lyon, Department of Cardiac Medicine, National Heart and Lung Institute, Dovehouse Street, London, SW3 6LY, UK; a.lyon{at}ic.ac.uk

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.

Keywords: adeno-associated virus; adenovirus; gene therapy; genetic vectors; myocardium

CiteULike    Complore    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this?

This article has been cited by other articles:

• Gupta, R., Tongers, J., Losordo, D. W. (2009). Human Studies of Angiogenic Gene Therapy. Circ. Res. 105: 724-736 [Abstract] [Full Text]  
• Huang, M., Chen, Z., Hu, S., Jia, F., Li, Z., Hoyt, G., Robbins, R. C., Kay, M. A., Wu, J. C. (2009). Novel Minicircle Vector for Gene Therapy in Murine Myocardial Infarction. Circulation 120: S230-S237 [Abstract] [Full Text]  
• Antoons, G., Sipido, K. R. (2008). Targeting calcium handling in arrhythmias. Europace 10: 1364-1369 [Abstract] [Full Text]  
• Edelman, R. R. (2008). Can MR Imaging Be Used to Determine the Efficacy of Gene Therapy in the Heart?. Radiology 249: 1-2 [Full Text]