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)
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Study Section
Special Emphasis Panel (ZDK1-GRB-W (M2))
Program Officer
Mckeon, Catherine T
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University of California San Francisco
Internal Medicine/Medicine
Schools of Medicine
San Francisco
United States
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Zhang, Hua; Verkman, A S (2014) Longitudinally extensive NMO spinal cord pathology produced by passive transfer of NMO-IgG in mice lacking complement inhibitor CD59. J Autoimmun 53:67-77
Veit, Guido; Avramescu, Radu G; Perdomo, Doranda et al. (2014) Some gating potentiators, including VX-770, diminish ?F508-CFTR functional expression. Sci Transl Med 6:246ra97
Esteva-Font, Cristina; Jin, Byung-Ju; Verkman, A S (2014) Aquaporin-1 gene deletion reduces breast tumor growth and lung metastasis in tumor-producing MMTV-PyVT mice. FASEB J 28:1446-53
Valentine, Cathleen D; Zhang, Hua; Phuan, Puay-Wah et al. (2014) Small molecule screen yields inhibitors of Pseudomonas homoserine lactone-induced host responses. Cell Microbiol 16:1-14
Asavapanumas, Nithi; Ratelade, Julien; Papadopoulos, Marios C et al. (2014) Experimental mouse model of optic neuritis with inflammatory demyelination produced by passive transfer of neuromyelitis optica-immunoglobulin G. J Neuroinflammation 11:16
Ye, Long; Hu, Bao; El-Badri, Faris et al. (2014) ?F508-CFTR correctors: synthesis and evaluation of thiazole-tethered imidazolones, oxazoles, oxadiazoles, and thiadiazoles. Bioorg Med Chem Lett 24:5840-4
Xie, Moses; Blackman, Brigitte; Scheitrum, Colleen et al. (2014) The upstream conserved regions (UCRs) mediate homo- and hetero-oligomerization of type 4 cyclic nucleotide phosphodiesterases (PDE4s). Biochem J 459:539-50
Tradtrantip, Lukmanee; Ko, Eun-A; Verkman, Alan S (2014) Antidiarrheal efficacy and cellular mechanisms of a Thai herbal remedy. PLoS Negl Trop Dis 8:e2674
Katada, Ryuichi; Akdemir, Gokhan; Asavapanumas, Nithi et al. (2014) Greatly improved survival and neuroprotection in aquaporin-4-knockout mice following global cerebral ischemia. FASEB J 28:705-14
Asavapanumas, Nithi; Verkman, A S (2014) Neuromyelitis optica pathology in rats following intraperitoneal injection of NMO-IgG and intracerebral needle injury. Acta Neuropathol Commun 2:48

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