CHAPTER 1 – Retroviral Vector Design for Cancer Gene Therapy

This chapter discusses the retroviral vector design for cancer gene therapy. Most of the failures of gene therapy trials can be attributed to a discordant combination of over interpreted clinical concepts and immature technology, including poor vector design. Nevertheless, many former skeptics were turned to true believers, not only due to the enormous public and economical interest to vector designs. An ideal vector should allow efficient and selective transduction of the target cell of interest be maintained, be expressed at levels necessary for achieving therapeutic effects, and be safe in terms of avoiding unexpected side effects in the host. Viruses are a perfect tool for gene transfer as they have evolved to deliver their genome efficiently to target cells with subsequent high-level gene expression. Vector systems for therapeutic gene transfer have been developed from different virus groups, each system having specific advantages and drawbacks. Retroviruses have several unique features that render them highly suitable for vector development.