The UNC MTCC Program has selected a configuration of six Cores and three Pilot & Feasibility Project to best synergize and accelerate the progress of molecular therapy research at UNC-CH. The Administrative Core (Core A, R.C. Boucher, P.I.) will oversee and coordinate all aspects of the MTCC operation, including the operation of Core facilities and the selection and oversight of the Pilot & Feasibility Program. In addition, this Core will interface with other important activities on the UNC-CH campus, including training and clinical trials networks. Each Core will provide novel and important services to the UNC-CH molecular therapeutics community. The Vector Core (Core B, D. McCarty, P.I.) will provide new vectors (high capacity adenoviral, double-stranded and chimeric AAV, paramyxovirus) and services (plasmid preparation). The Molecular Core (Core C, W. O'Neal, P.I.) will provide a mouse with CF-like lung disease (the betaENaC mouse) and toxicogenomics capabilities. The Imaging & Histology Core (Core D, C.W. Davis, P.I.) will provide a series of important new imaging technologies, as well as histology and EM capabilities. The Correction Core (Core E, S.E. Gabriel, P.I.) will provide UNC-CH investigators with a wide range of assays to detect correction of CF defects both in in vivo and in vitro systems. The Cell Culture Models Core (Core F, S. Randell, P.I.) will provide additional cell types to the MTCC community, e.g., microvascular endothelial cells and hepatocytes, as well as novel airway culture systems that reprise the in vivo condition. The Cores will be supplemented by three Pilot & Feasibility Projects. P&F 1 (R. Pickles, P.I.), in collaboration with Dr. Mark Peeples at Rush University, will explore the feasibility of generating non-cytotoxic, RSV-replicon/ viruses as prototype gene transfer vectors for airway epithelia. P&F 2 (D. McCarty, P.I.) will develop novel AAV serotypes and hybrid serotypes for efficient gene transfer. P&F 3 (T. Kafri, P.I.) will explore a novel technology to generate efficient production of transgenic animals as models for gene transfer researchers. The MTCC is complemented by an environment rich in basic research, clinical research, and training capacity that should guarantee maximal utilization of this program.
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