Gene therapy and CVD: How near are we?

Critical for success of any gene therapy approach is the efficient packaging, effective cell specific delivery and nuclear translocation of the nucleic acid with minimal toxicity. Delivery systems utilizing a wide variety of viral vectors have traditionally been used to modify genomic DNA. However, drawbacks to the viral vectors include difficulties in large-scale production, potential contamination by wild-type viral particles and immunogenicity. Thus, efficient non-viral delivery of both plasmids for transgene expression and short oligonucleotides for modulating cellular functions has been developed. Gene therapy is now a consideration in the treatment of certain inherited and acquired genetic disorders associated with cardiovascular disease (CVD). Furthermore, many other cardiovascular conditions are potential targets for gene therapy, and advances in knowledge will increase the ability to link specific genes to a disease, resulting in the identification of further targets. With improvements in delivery and targeting, gene therapy is likely to substantially augment established and emerging therapies in reducing the global burden of cardiovascular disease.