Goals of Program The major goal of this Center is the development of effective gene therapies for the treatment of metabolic diseases, with a focus on CF. The Center has helped to facilitate this goal through several mechanisms, of which the centralized Core Facilities and the Pilot and Feasibility Program are among the most significant. The purpose of this Pilot Program is to provide seed money for research that has not yet reached the stage of maturity necessary for funding through traditional NIH mechanisms such as R01 and P01 grants, but is considered to be innovative and potentially high risk. The program is also committed to assisting junior investigators in the development of their careers, to the point at which they can establish independent laboratories with independently funded research programs in Molecular Medicine. Additionally, this program has served as a conduit to recruit new senior investigators into the field of gene therapy by promoting interdisciplinary interactions with the Center's Cores and established Center Members. Ultimately, the success of this program will be judged on the ability of these pilot projects to mature into fundable NIH proposals, as well as on the placement of junior, non-tenure-track, faculty into tenure-track faculty positions with independent laboratories.
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