GENE DELIVERY SYSTEMS FOR USE IN GENE-THERAPY - AN OVERVIEW OF QUALIFY ASSURANCE AND SAFETY ISSUES

The development of safe and effective agents for gene therapy is found ed on three main principles; careful choice and design of vectors, ass essment of vector safety under GLP and production of the vector stocks under GMP. The first ensures the safe and appropriate contained deliv ery and expression of the required gene to the recipient of the therap y. GLP provides fully documented studies of potency, efficacy and safe ty of the product while the production of clinical grade agents under GMP is essential.