The development of effective gene therapies for inherited disorders requires interdisciplinary programs that focus on disease gene identification, disease pathogenesis, and the development of technologies for gene transfer. This Center's goal has been to provide investigators with the opportunity to improve and/or expand their gene therapy-based research. Although this Center has a primary focus on the development of gene therapies for cystic fibrosis (CF), as evidenced through two of its specialized Research Cores that apply specialized airway model systems to CF research, it also maintains an active research program in molecular medicine to investigate a diverse range of other genetic diseases. The Center has productively fostered gene therapy research through the following mechanisms. 1) The Center's Pilot and Feasibility Program that has sponsored 33 pilots over the previous funding period and has brought numerous new members and expertise into the Center while fostering the development of talented junior Associate Members into independent tenure-track faculty. 2) The Center has successfully established or expanded existing specialized Core facilities (Vector Core, Cell and Tissue Core, Morphology Core and Animal Models Core) devoted to gene therapy research. These Cores have provided investigators with specialized vectors, human and mouse CF model systems, and methods that have allowed them to test hypotheses that could not otherwise be evaluated. 3) The Center has successfully instituted a CF Lung Tissue Acquisition Consortium with nine outside lung transplant centers that has allowed for significant expansion of use of the humanized CF model systems in the Cells and Tissue Core and Animal Models Core by Center Members. This consortium, which receives between 15-20 CF lung transplants per year has also promoted numerous intra-institutional collaborations on CF research that have also spawned the development of new research programs. 4) The Center has maintained an active Enrichment Program consisting of a Centralized Center Web site, a trainee seminar series, external speaker seminar series, informal smaller research focus groups, and an annual Gene Therapy Center Retreat. All of these programs have significantly enhanced interdisciplinary interactions between Center members and the education of the lay public in areas of gene therapy. 5) The Center has also significantly expanded the use and development of gene vector systems not only within it member laboratories but also in collaboration with four companies and members of the CF Lung Tissue Acquisition Consortium. 6) The Center has established 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 has greatly strengthened existing gene therapy-based research programs at the University of Iowa and Consortium Institutions, leading to the development of numerous basic and applied research findings that have enhanced the utility of gene therapies to both study and treat genetic diseases.
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