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Introduction

Marsico Lung Institute/UNC Cystic Fibrosis Center

Introduction

The Cystic Fibrosis and Pulmonary Diseases Research and Treatment Center (CF Center) at the University of North Carolina at Chapel Hill traditionally has had a single goal: to “cure” CF lung disease. To this end, we assembled a broad-based biomedical research team to tackle this goal. Thus, we have wide-ranging skills in ion transport physiology (Ussing chamber, patch clamp, confocal surface liquid imaging), mucus cell/mucin secretion biology and biochemistry, transgenic and gene-targeted mouse models, in vivo measures of mucociliary clearance and measures of mucin concentration/hydration, and Phase I clinical trials. In addition, we have added expertise focused on specific CF microbiologic problems of the lung, including Pseudomonas aeruginosa, anaerobic bacteria, and now Burkholderia cepacia. These efforts are complemented by a major effort to identify genetic modifiers of all facets of CF airways pathogenesis. Thus, the airways pathobiology/therapeutics efforts have produced multiple collaborations within the UNC MLI, multiple national/international collaborations, and, indeed, collaboration with the CFF Mucus Consortium and Gene Modifier groups.

With the arrival on the UNC campus of Dr. Jack Riordan and his group and Dr. Doug Cyr, we have become a more broad and ‘mature’ CF Center, i.e., we have developed as a second goal: to define the abnormalities in mutant CFTR biogenesis/trafficking, to develop the appropriate primary screens and secondary screens for mutant CFTR “potentiator/corrector” hit/lead evaluation, and to refine the clinical biophysical and biochemical assays needed for “potentiator/corrector” drug development. Again, this effort has melded together collaborative UNC CF Center groups focused on CF genetics, CFTR biochemistry and cell biology, high throughput drug screening paradigms, animal models of CF, and clinical development/validation of both nasal and rectal biophysical and biochemical analyses of correction of mutant CFTR processing defects.

Thus, the UNC CF Center has now been organized to provide the maximum technical and intellectual support to both goals, with a common focus on the rapid “translation” of basic science insights into the clinical patient care arena. The centerpiece of the UNC CF Center is the network of Core Laboratories that underpin all CF research at UNC. As a group, the Cores operate under two common themes. First, the Cores provide access for UNC CF investigators to a broad range of quality-controlled, diverse reagents and technologies. Second, the Cores provide a strong intellectual/research contribution to the UNC CF Center by virtue of the fact that the Cores are led by talented and productive scientists. Indeed, our three major Cores (Tissue Procurement and Cell Culture Core, Molecular Biology Core, and Animal Models Core) have become resources to universities both nationally and internationally, and indeed to CFFTI and industry collaborators, to make available reagents and expertise to CF investigators in need of cellular, molecular, and mouse reagents.

The CF Center achieves its purpose through:

  • Core laboratories that provide training, technical support, laboratory animals, or assays;

  • A pilot/feasibility program that offers startup funds to junior investigators or to established investigators who wish to pursue new directions in research;

  • A scientific enrichment program that improves the intellectual climate for gastrointestinal biological research and promotes cooperation, collaboration, and communication among involved personnel; and

  • A professional development and training program that promotes the development of junior faculty.

-Dr. Richard C. Boucher, Director of the Marsico Lung Institute-CF Center