FOREFRONTS IN NEPHROLOGY SYMPOSIUM--GENE THERAPY

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DESCRIPTION (adapted from the application) Recent advances in our understanding of the molecular basis of renal disease provide the potential for developing molecular based strategies for therapy. They include the cloning of the "minor" collagens and the discovery of their role in Goodpasture's and Alport syndrome, the cloning of transport proteins in the kidney that are involved with Gitelman's and Bartter's syndromes, and the identification of the genetic defect associated with PKD 1 and 2. Recent reports of linkage in families with FSGS and IgA nephropathy and the identification of quantitative trait loci that affect the phenotypic expression of cyst formation in the PKD mouse have provided new insights into the molecular basis of renal pathogenesis. The evidence from angiotensinogen knock-outs and quantitative "knock-in" experiments has provided proof of concept and molecular evidence that additive genetic traits may control long-term blood pressure regulation by the kidney. How these findings will translate into better therapies for patients remains to be determined but is clearly a long-term goal. This application requests partial support for the costs of a conference that will help facilitate entry of investigators into this area in renal research entitled, "Frontiers in Nephrology, Gene Therapy." This conference, to be held June 4-6 in Beaver Creek Colorado, will be a satellite meeting that immediately follows the American Society of Gene Therapy in Denver, Colorado. The goal of the conference is to bring leaders in gene therapy and molecular genetics together with trainees and junior faculty to provide an environment that will promote interaction and facilitate future research in this field. The hope is to bring the two fields together to address issues of importance to the renal community. The meeting will be held in a single room conference in the style of a Keystone or Gordon Conference and poster sessions will provide additional opportunities for the attendees. The conference will be advertised in Science, Nature Medicine, Kidney International, and Journal of Molecular Therapy. In addition, announcements will be made at the ASN in Miami and brochures will be provided both there and will be sent to program directors as well.