Gene delivery systems for use in gene therapy: an overview of quality assurance and safety issues

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