VECTOR DEVELOPMENT - A MAJOR OBSTACLE IN HUMAN GENE-THERAPY

Gene therapy has been proposed for a wide variety of human conditions including monogenic disorders, such as the haemoglobinopathies and imm unodeficiency syndromes, cancer and many other diseases. Prerequisites for the success of this approach include the ability to deliver the t herapeutic gene intact to the target cell, persistent levels of transg ene expression sufficient to correct the disease phenotype, lack of un wanted side-effects associated with vector exposure or gene transfer a nd relative simplicity allowing the widespread use of this methodology . Although substantial progress has been made in animal models since t he inception of genetic therapy in the early 1980s, significant obstac les remain for human therapy, most notably in the area of vector devel opment. The first generation of gene therapy vectors has failed to ove rcome many of the biological hurdles cited above necessitating the dev elopment of alternate means of gene delivery and expression.