Nonviral vectors for cardiovascular gene delivery.

Abstract

The development of artificial gene delivery systems is an attractive problem. The delivery of nucleic acids to cells initially grew out of basic studies in molecular biology and virology but is now mainly considered within the context of gene therapy. This new therapeutic approach promises to be a singular advance in the treatment of both acquired and genetic diseases at the most fundamental levels of pathology. Nucleic acids are transferred into target cells to modify their genetic instructions so as to ameliorate or prevent a disease. Yet, the challenging problem of efficiently transferring and stably expressing transgenes in appropriate tissues needs to be solved in order for the great promise of gene therapy to be realized. One approach, viral vectors, is the basis of most preclinical studies and human clinical trials. Another approach is artificial gene delivery systems that go by a variety of descriptive names such as synthetic delivery systems, physical-chemical methods, and non-viral vectors, each of which emphasize different aspects of the approach. Table 1 lists the types of synthetic delivery methods that are under development.