Gene therapy: targeting the myocardium.
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.
Lyon
A R
AR
Department of Cardiac Medicine, National Heart and Lung Institute, Dovehouse Street, London, SW3 6LY, UK. a.lyon@ic.ac.uk
Sato
M
M
Hajjar
R J
RJ
Samulski
R J
RJ
Harding
S E
SE
D009202:Cardiomyopathies / Q000628:therapy*; D000229:Dependovirus / Q000235:genetics; D015967:Gene Expression Regulation, Viral; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D017410:Practice Guidelines as Topic; D012190:Retroviridae / Q000235:genetics*