The goal of this application is to support the second Cold Spring Harbor meeting on Human Gene Therapy. The first meeting in this new series, organized by two of the present applications (T. Friedmann and R. Mulligan) and W.F. Anderson, was held in September 1992 and attracted a total of 300 scientists. Since that meeting, a great deal of progress has been made in the development of gene transfer methods, the characterization of appropriate human disease targets and animal models, and in results from the more than 60 human studies approved by the Recombinant DNA Advisory Committee of the NIH and the FDA. The rapid pace of progress in this field calls for continuing evaluation and information exchange as is done so effectively at meetings sponsored by the Cold Spring Harbor Laboratories. Dr. Y.Y. Kan will join Drs. Friedmann and Mulligan in organizing the 1994 meeting which will include sessions on vectors and gene transfer, cell and organ transplantation, in vivo gene expression, animal models, human clinical studies and new and developing technology.