ABSTRACT
Over the past few decades, our knowledge of the molecular and genetic basis of disease has dramatically increased, which, combined with advances in technology, has resulted in novel molecular therapies for disease. In particular, gene therapy, which involves the transfer of genetic material to cells in order to produce a therapeutic effect, has become a promising approach, with 1145 clinical trials involving more than 3500 individuals currently being carried out worldwide (Journal of Gene Medicine, www.wiley.co.uk/wileychi/genmed). Initially, gene therapy was considered for monogenic disorders such as cystic fibrosis, in which one defective gene could be replaced by a functional gene. Indeed, the first gene therapy clinical trial was for adenosine deaminase deficiency,1 and we have almost reached a point at which single-gene disorders have
been cured. However, it is now recognized that gene therapy represents an effective approach for more complex diseases such as cancer, either as single agents or in conjunction with chemotherapy or radiotherapy.2 Cancer gene therapy represents one of the fastest growing areas of pre-clinical and clinical cancer research. Out of the 1145 gene therapy clinical trials being undertaken, 762 are in cancer gene therapy (Table 13.1). This chapter discusses the current strategies for cancer gene therapy in both preclinical and clinical settings, as well as the vectors used to deliver the genes to the target cells.
