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
Mckeon, Catherine T
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University of North Carolina Chapel Hill
Internal Medicine/Medicine
Schools of Medicine
Chapel Hill
United States
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Duncan, Gregg A; Kim, Namho; Colon-Cortes, Yanerys et al. (2018) An Adeno-Associated Viral Vector Capable of Penetrating the Mucus Barrier to Inhaled Gene Therapy. Mol Ther Methods Clin Dev 9:296-304
Gentzsch, Martina; Mall, Marcus A (2018) Ion Channel Modulators in Cystic Fibrosis. Chest 154:383-393
Terryah, Shawn T; Fellner, Robert C; Ahmad, Saira et al. (2018) Evaluation of a SPLUNC1-derived peptide for the treatment of cystic fibrosis lung disease. Am J Physiol Lung Cell Mol Physiol 314:L192-L205
Gillen, Austin E; Yang, Rui; Cotton, Calvin U et al. (2018) Molecular characterization of gene regulatory networks in primary human tracheal and bronchial epithelial cells. J Cyst Fibros 17:444-453
Muhlebach, Marianne S; Zorn, Bryan T; Esther, Charles R et al. (2018) Initial acquisition and succession of the cystic fibrosis lung microbiome is associated with disease progression in infants and preschool children. PLoS Pathog 14:e1006798
Cholon, Deborah M; Gentzsch, Martina (2018) Recent progress in translational cystic fibrosis research using precision medicine strategies. J Cyst Fibros 17:S52-S60
Porrello, Alessandro; Leslie, Patrick L; Harrison, Emily B et al. (2018) Factor XIIIA-expressing inflammatory monocytes promote lung squamous cancer through fibrin cross-linking. Nat Commun 9:1988
Trimble, Aaron T; Whitney Brown, A; Laube, Beth L et al. (2018) Hypertonic saline has a prolonged effect on mucociliary clearance in adults with cystic fibrosis. J Cyst Fibros 17:650-656
Panganiban, Ronald A; Sun, Maoyun; Dahlin, Amber et al. (2018) A functional splice variant associated with decreased asthma risk abolishes the ability of gasdermin B to induce epithelial cell pyroptosis. J Allergy Clin Immunol 142:1469-1478.e2
Muhlebach, Marianne S; Hatch, Joseph E; Einarsson, Gisli G et al. (2018) Anaerobic bacteria cultured from cystic fibrosis airways correlate to milder disease: a multisite study. Eur Respir J 52:

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