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).
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.
|Wang, Wei; Hong, Jeong S; Rab, Andras et al. (2016) Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators. PLoS One 11:e0152232|
|Mutyam, Venkateshwar; Libby, Emily Falk; Peng, Ning et al. (2016) Therapeutic benefit observed with the CFTR potentiator, ivacaftor, in a CF patient homozygous for the W1282X CFTR nonsense mutation. J Cyst Fibros :|
|Wei, Shipeng; Roessler, Bryan C; Icyuz, Mert et al. (2016) Long-range coupling between the extracellular gates and the intracellular ATP binding domains of multidrug resistance protein pumps and cystic fibrosis transmembrane conductance regulator channels. FASEB J 30:1247-62|
|Ehrhardt, Annette; Chung, W Joon; Pyle, Louise C et al. (2016) Channel Gating Regulation by the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) First Cytosolic Loop. J Biol Chem 291:1854-65|
|Roy, Bijoyita; Friesen, Westley J; Tomizawa, Yuki et al. (2016) Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression. Proc Natl Acad Sci U S A 113:12508-12513|
|Mutyam, Venkateshwar; Du, Ming; Xue, Xiaojiao et al. (2016) Discovery of Clinically Approved Agents That Promote Suppression of Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations. Am J Respir Crit Care Med 194:1092-1103|
|Bali, Vedrana; Lazrak, Ahmed; Guroji, Purushotham et al. (2016) Mechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic Fibrosis. PLoS One 11:e0155882|
|Veit, Gudio; Avramescu, Radu G; Chiang, Annette N et al. (2016) From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations. Mol Biol Cell 27:424-33|
|Solomon, George M; Raju, S Vamsee; Dransfield, Mark T et al. (2016) Therapeutic Approaches to Acquired Cystic Fibrosis Transmembrane Conductance Regulator Dysfunction in Chronic Bronchitis. Ann Am Thorac Soc 13 Suppl 2:S169-76|
|Bali, Vedrana; Lazrak, Ahmed; Guroji, Purushotham et al. (2016) A synonymous codon change alters the drug sensitivity of Î”F508 cystic fibrosis transmembrane conductance regulator. FASEB J 30:201-13|
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