Pilot and Feasibility Projects at our Institution help establish multidisciplinary collaborations between basic and translational scientists, and integrate well within the overall CF Center. The projects described in this component and associated illustrations reinforce central topics pursued by the P30, including CFTR cellular and structural biology, mechanisms underlying CF tissue pathogenesis, and novel therapeutic interventions for the disease. Pilots are intended to provide initial support for innovative directions and stimulate investigators from other areas to lend their expertise to research concerning CF. The P30 Pilot Program has allowed Center leadership to identify and fund promising early stage research. Functions/goals of this Component are identified by the following Specific Aims:
Specific Aim 1 ? Provide research support that will enable eligible investigators to explore the feasibility of innovative concepts. Projects typically last 1-2 years each, are concordant with the overall objectives of the UAB P30, and are intended to result in further grant support from NIH or other funding agencies.
Specific Aim 2. Provide an administrative framework for oversight and review of Pilot and Feasibility Studies. This includes recommendations regarding continuation (or termination) of Pilots to the P30 Internal Advisory Committee, solicitation and review of Pilot applications, record-keeping with regard to grant and manuscript productivity of Pilot PIs, subsequent career events of awardees, and all aspects of program management. During the current funding cycle, the Pilot and Feasibility Component has been successful as judged by the following criteria: 1) funds were well utilized and awards made to investigators meeting eligibility criteria;2) projects were focused on P30 Center scientific priorities;and 3) Pilot and Feasibility studies were productive (e.g. in terms of publications, subsequent independent R01 or other peer-reviewed support, and/or attraction of new investigator into Center-related research).

Public Health Relevance

From a historical standpoint, Pilot resources have assisted several members of the CF Research Base early in their scientific careers. The Pilot mechanism also helped launch careers of investigators who subsequently accepted faculty positions at outside institutions and continue to pursue CF research. At just four years into its first funding cycle, the UAB P30 Pilot Component has already made important contributions to understanding cystic fibrosis disease mechanism and translational research.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Center Core Grants (P30)
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University of Alabama Birmingham
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Heltshe, Sonya L; Rowe, Steven M; Mayer-Hamblett, Nicole (2014) Evaluating the predictive ability of sweat chloride. J Cyst Fibros 13:118
Hill, Aubrey E; Plyler, Zackery E; Tiwari, Hemant et al. (2014) Longevity and plasticity of CFTR provide an argument for noncanonical SNP organization in hominid DNA. PLoS One 9:e109186
Dean, Nichole; Ranganath, Neel K; Jones, Brandon et al. (2014) Porcine nasal epithelial cultures for studies of cystic fibrosis sinusitis. Int Forum Allergy Rhinol 4:565-70
Lee, Seakwoo; Henderson, Mark J; Schiffhauer, Eric et al. (2014) Interference with ubiquitination in CFTR modifies stability of core glycosylated and cell surface pools. Mol Cell Biol 34:2554-65
Oren, Yifat S; McClure, Michelle L; Rowe, Steven M et al. (2014) The unfolded protein response affects readthrough of premature termination codons. EMBO Mol Med 6:685-701
Xue, Xiaojiao; Mutyam, Venkateshwar; Tang, Liping et al. (2014) Synthetic aminoglycosides efficiently suppress cystic fibrosis transmembrane conductance regulator nonsense mutations and are enhanced by ivacaftor. Am J Respir Cell Mol Biol 50:805-16
McClure, Michelle L; Wen, Hui; Fortenberry, James et al. (2014) S-palmitoylation regulates biogenesis of core glycosylated wild-type and F508del CFTR in a post-ER compartment. Biochem J 459:417-25
Zhang, Shaoyan; Ranganath, Neel K; Skinner, Daniel et al. (2014) Marked repression of CFTR mRNA in the transgenic Cftr(tm1kth) mouse model. J Cyst Fibros 13:351-2
Boyle, Michael P; Bell, Scott C; Konstan, Michael W et al. (2014) A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med 2:527-38
Tuggle, Katherine L; Birket, Susan E; Cui, Xiaoxia et al. (2014) Characterization of defects in ion transport and tissue development in cystic fibrosis transmembrane conductance regulator (CFTR)-knockout rats. PLoS One 9:e91253

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