This is a proposal to continue our Cystic Fibrosis (CF) Research and Translational Core Center at the University of California, San Francisco and collaborating institutions. The focus of our Core Center remains the discovery and evaluation of novel small-molecule therapies for CF. The original proposal followed from 5 years of work establishing a unique academic drug discovery program in the laboratories of Dr. Verkman and collaborators to identify and characterize small-molecule modulators of CFTR activity. The proposed Core Center will fund five Cores to support the activities of 2 Pilot Projects and 28 CF-related projects. The Core directors are experienced CF investigators with recognized expertise in their areas of investigation and a history of productive collaboration. The Cores include: High-Throughput Screening (Core A, Alan Verkman, director), Clinical Resources (Core B, Dennis Nielson, director), Cell Models (Core C, Walter Finkbeiner, director), Synthetic Chemistry (Core D, Mark Kurth, director), and Cell &Tissue Bioassays (Core E, Peter Haggie, director). Projects to utilize the cores include the discovery of modulators of epithelial ion transporters (CFTR, CaCCs, ENaC, NKCC, potassium channels), epithelial cell mucin secretion, and Pseudomonas biofilm formation. Other projects to utilize the cores include research on mechanisms of lung disease in CF. The general goal of the research to be enhanced by the Core Center is to develop new small-molecule therapies for CF that can be translated into the clinic.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Center Core Grants (P30)
Project #
Application #
Study Section
Special Emphasis Panel (ZDK1-GRB-W (M2))
Program Officer
Mckeon, Catherine T
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
University of California San Francisco
Internal Medicine/Medicine
Schools of Medicine
San Francisco
United States
Zip Code
Piechowicz, Katarzyna A; Truong, Eric C; Javed, Kashif M et al. (2016) Synthesis and evaluation of 5,6-disubstituted thiopyrimidine aryl aminothiazoles as inhibitors of the calcium-activated chloride channel TMEM16A/Ano1. J Enzyme Inhib Med Chem 31:1362-8
Bonser, Luke R; Zlock, Lorna; Finkbeiner, Walter et al. (2016) Epithelial tethering of MUC5AC-rich mucus impairs mucociliary transport in asthma. J Clin Invest 126:2367-71
Haggie, Peter M; Phuan, Puay-Wah; Tan, Joseph-Anthony et al. (2016) Inhibitors of pendrin anion exchange identified in a small molecule screen increase airway surface liquid volume in cystic fibrosis. FASEB J 30:2187-97
Suzuki, Shingo; Sargent, R Geoffrey; Illek, Beate et al. (2016) TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs. Mol Ther Nucleic Acids 5:e273
Flores, Alyssa M; Casey, Scott D; Felix, Christian M et al. (2016) Small-molecule CFTR activators increase tear secretion and prevent experimental dry eye disease. FASEB J 30:1789-97
Cil, Onur; Haggie, Peter M; Phuan, Puay-Wah et al. (2016) Small-Molecule Inhibitors of Pendrin Potentiate the Diuretic Action of Furosemide. J Am Soc Nephrol 27:3706-3714
Jin, Byung-Ju; Smith, Alex J; Verkman, Alan S (2016) Spatial model of convective solute transport in brain extracellular space does not support a ""glymphatic"" mechanism. J Gen Physiol 148:489-501
Cil, Onur; Phuan, Puay-Wah; Lee, Sujin et al. (2016) CFTR activator increases intestinal fluid secretion and normalizes stool output in a mouse model of constipation. Cell Mol Gastroenterol Hepatol 2:317-327
Esteva-Font, Cristina; Jin, Byung-Ju; Lee, Sujin et al. (2016) Experimental Evaluation of Proposed Small-Molecule Inhibitors of Water Channel Aquaporin-1. Mol Pharmacol 89:686-93
Walentek, Peter; Quigley, Ian K; Sun, Dingyuan I et al. (2016) Ciliary transcription factors and miRNAs precisely regulate Cp110 levels required for ciliary adhesions and ciliogenesis. Elife 5:

Showing the most recent 10 out of 241 publications