American Society of Gene Therapy - Third Annual Meeting.

: The field of gene therapy, delivering genes to directly treat diseases, has had a remarkable year. This is no more evident than in the scope of the third annual meeting of the American Society of Gene Therapy (ASGT). Clear progress has been made in both ex vivo clinical protocols and in vivo administration. The meeting covered every major method of gene delivery, from injection of naked DNA to advanced synthetic gene delivery systems, as well as the major viral-based vectors. The optimism of the society was tempered, however, by the much-publicized death of a patient in a clinical trial at the University of Pennsylvania last year. There was a correspondingly high regulatory presence at the meeting, with several presentations by representatives of the US FDA and National Institutes of Health (NIH). Major clinical advances in gene therapy have been in genetic diseases, including hemophilia, severe combined immunodeficiency, and cystic fibrosis. Therapies are in later-stage clinical trials, and evidence of efficacy has been demonstrated, most notably by the apparent cure of SCID-affected children in Paris by ex vivo gene therapy with cytokine receptor subunit genes. Cancer gene therapy is also making significant headway, with many products entering phase II and III trials. Basic technology development is proceeding in vector targeting, enhancement of gene transfer efficiency, and regulating expression of therapeutic genes. In addition, basic research demonstrates the promise of new combined modes for treating diseases such as muscular dystrophy, lysosomal storage diseases and cardiovascular disease.