Hybrid Adenoviral Vectors

The characterization of disease at the genetic level facilitates potential genotypic and/or phenotypic correction by gene therapy. Although the concept of gene therapy has been extensively established over the past two decades, the development of effective clinical protocols to facilitate efficacious reversal of disease has proven highly problematic. The development of an effective gene delivery system to the site of therapeutic significance has proven to be the major hurdle to the advancement of gene therapies. Many questions currently remain unanswered and these raise major debates over the best vector systems to treat a specific clinical disorder, and at a more fundamental level the choice of gene to be applied. The ultimate goal of a gene therapy protocol is the efficient targeted delivery of a therapeutic transgene, whose expression can be sufficiently regulated, in a defective tissue. Vector delivery would ideally involve a single, lifetime treatment by a simple, non-invasive, and safe protocol, which can be incorporated into clinical practice. The vast array of clinical diseases, for which gene therapy presents clinical promise, demands a multitude of different requirements for a vector system to meet.