The UNC MTCC Program has selected a configuration of six Cores and three Pilot &Feasibility Project to 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, J. Beecham, P.I.) will provide new vectors (double-stranded, shuffled, chimeric AAV, high capacity adenoviral, paramyxovirus) and services (plasmid preparation). The Molecular Biology and Mouse Core (Core C, W. O'Neal, P.I.) will provide mice with CF like lung disease ((3ENaC and pENaC/CF mice) and shRNA technologies. The Imaging Core (Core D, C.W. Davis, P.I.) will provide a range of 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 airway and G.I. assays to detect correction of CF defects both in in vivo and in vitro systems. The Cell Models Core (Core F, S. Randell, P.I.) will provide additional cell types to the MTCC community, e.g., airway and G.I. stem cells, as well as novel airway immortalized cell lines. The Cores will be supplemented by three Pilot &Feasibility Projects. P&F 7 (L. Ostrowski, P.I.), in collaboration with Drs. Olsen and Kafri, will explore the efficiency requirements for correcting with lentiviruses diseases characterized as "ciliopathies". P&F 8 (A. Asokan, P.I.), in collaboration with Dr. Samulski, will explore the targeting of G.I. epithelia, including stem cells, for CF mouse gut correction. P&F 9 (J. Olsen, P.I.) will, in collaboration with Dr. Grubb, test the efficiency of integrating vs. non-integrating EIAV lentiviral vectors in correcting the neonatal phenotype of the CF-like (3ENaC/AF508 CF mouse lung. The MTCC is complemented at UNC by an environment rich in basic science, translational research, and training that will guarantee maximal utilization of MTCC resources.

Public Health Relevance

Cystic fibrosis is the most common lethal genetic disease affecting Caucasian Americans. The efforts of the MTCC are dedicated towards developing gene therapy technologies designed to arrest or even cure this disease.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Center Core Grants (P30)
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Study Section
Special Emphasis Panel (ZDK1-GRB-1 (J2))
Program Officer
Eggerman, Thomas L
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University of North Carolina Chapel Hill
Internal Medicine/Medicine
Schools of Medicine
Chapel Hill
United States
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Ostrowski, L E; Yin, W; Patel, M et al. (2014) Restoring ciliary function to differentiated primary ciliary dyskinesia cells with a lentiviral vector. Gene Ther 21:253-61
Morales Johansson, Helena; Newman, Donna R; Sannes, Philip L (2014) Whole-genome analysis of temporal gene expression during early transdifferentiation of human lung alveolar epithelial type 2 cells in vitro. PLoS One 9:e93413
Henderson, Ashley G; Ehre, Camille; Button, Brian et al. (2014) Cystic fibrosis airway secretions exhibit mucin hyperconcentration and increased osmotic pressure. J Clin Invest 124:3047-60
Saini, Yogesh; Dang, Hong; Livraghi-Butrico, Alessandra et al. (2014) Gene expression in whole lung and pulmonary macrophages reflects the dynamic pathology associated with airway surface dehydration. BMC Genomics 15:726
Esther Jr, Charles R; Boucher, Richard C; Johnson, M Ross et al. (2014) Airway drug pharmacokinetics via analysis of exhaled breath condensate. Pulm Pharmacol Ther 27:76-82
Cholon, Deborah M; Quinney, Nancy L; Fulcher, M Leslie et al. (2014) Potentiator ivacaftor abrogates pharmacological correction of ?F508 CFTR in cystic fibrosis. Sci Transl Med 6:246ra96
Tadokoro, Tomomi; Wang, Yang; Barak, Larry S et al. (2014) IL-6/STAT3 promotes regeneration of airway ciliated cells from basal stem cells. Proc Natl Acad Sci U S A 111:E3641-9
Bove, Peter F; Dang, Hong; Cheluvaraju, Chaitra et al. (2014) Breaking the in vitro alveolar type II cell proliferation barrier while retaining ion transport properties. Am J Respir Cell Mol Biol 50:767-76
Guo, Xueliang; Zheng, Shuo; Dang, Hong et al. (2014) Genome reference and sequence variation in the large repetitive central exon of human MUC5AC. Am J Respir Cell Mol Biol 50:223-32
Schwab, Ute; Abdullah, Lubna H; Perlmutt, Olivia S et al. (2014) Localization of Burkholderia cepacia complex bacteria in cystic fibrosis lungs and interactions with Pseudomonas aeruginosa in hypoxic mucus. Infect Immun 82:4729-45

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