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The first conference‐course on vectorology: training young researchers to implement gene therapy

This supplement of The Journal of Gene Medicine brings together 20 review articles contributed by the speakers at the First Multidisciplinary Theoretical and Practical Training Course in Vectorology, held at Généthon in Evry (France) on April 14–26, 2002. This event was the first of a series of training initiatives on gene and cell therapy for the European Community that Généthon has developed and co-ordinated with other specialised centres. We initiated this project because we believe that the scientific and technological problems facing these new biomedical areas need to be re-examined today. Fifteen years of gene transfer research have generated an enormous amount of novel data leading to substantial progress in our fundamental knowledge of living cells. Combined with the recent availability of the complete human genome sequence, this knowledge has contributed to our understanding of how molecular alterations at the genetic and/or protein levels can cause human illness. However, critical evaluation of several hundred clinical trials reveals that gene therapy still has some way to go before it can be considered a medical science providing true benefit for patients. This is partly due to a lack of optimal coordination of effort between the various players: research laboratories, hospitals, biotechnology companies and regulatory agencies. In addition, knowledge of the basic biology of vectors has probably been insufficient to devise efficient gene transfer strategies, especially those using complex molecular entities such as viral vectors. Indeed, vectors currently remain at the prototype stage and are evolving towards therapeutic products in a somewhat haphazard fashion. Depending on their complexity, they are the object of varying degrees of technological implementation from the standpoints of industrial development, bio-safety assessment and regulations. In addition to our inadequate knowledge of the basic biology of vectors, another limiting step in the development of clinically useable products is the difficulty in producing vectors in quantities sufficient for thorough pre-clinical testing. For example, to obtain the quantity of lentiviral vector required to perform a simple liver gene therapy experiment in mice, important resources have to be mobilised, rendering the cost of these vectors prohibitive for many laboratories, and, more