ABSTRACT
Gene therapy has potential for the treatment of genetic disease, infectious disease, cancer and many others. The premises upon which this field are based are now being developed. To rationally move forward, however, it is critical that concepts which are set forth in the basic research arena using animal models are validated and tested in human clinical studies. A university medical center is an ideal place to test the concepts of gene therapy since basic researchers are in close proximity to clinical practice. However, the production of clinical lots of drug substance for human clinical studies is, for the most part, performed within the pharmaceutical or biotechnology industry. We describe here a specialized university laboratory for the production of clinical materials used in somatic cell or gene therapy.
