Development of effective gene therapies for inherited disorders requires interdisciplinary programs which focus on disease gene identification, disease pathogenesis, and the development of technologies for gene transfer. The goal of this proposal is to establish a Center that will provide investigators with the opportunity to improve and/or expand their gene therapy-based research. The primary focus of this Center will be on the development of gene therapies for cystic fibrosis (CF), but will also include several other genetic diseases. Several mechanisms are proposed to meet the goals of this Center. 1) The Center will support pilot and feasibility studies directed at CF and other genetic diseases. This will bring new members and new expertise into the Center. It also provides established investigators with the opportunity to pursue novel, high risk strategies for gene therapy. 2) The Center will strengthen relevant existing Cores (Vector Core and Cell Culture Core), and develop two new Cores (Morphology Core and Animal Models Core). These Cores provide investigators with specialized vectors, primary cultures of airway epithelia, various transformed cell lines, resources, and new methods that allow them to test hypotheses that could not otherwise be evaluated. 3) The Center has formulated a Midwest Regional Consortium for the acquisition CF lung transplant tissue. This consortium will create a repository of CF and non-CF primary airway cells and tissue which are critical for the gatherings. This provides a forum for critical evaluation of research directed at the development of new approaches, model systems, and novel ideas. 5) The Center will allow expansion of gene transfer research to newly characterized genetic diseases. This will take advantage of opportunities to develop new treatments in many areas. Translation of the work to other systems will also benefit the CF research effort. 6) The Center will establish formal internal mechanisms and external consultants to review the Center, the Cores, and the pilot and feasibility projects, thereby ensuring a high level of excellence and the most appropriate utilization of the Center's resources. In summary, the Center for Gene Therapy of CF and Other Genetic Diseases will greatly strengthen existing gene therapy-based research programs at the University of Iowa and will allow progression of the research in existing new directions.
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