(Overall for Pilot and Feasibility Program) The Pilot and Feasibility Program, directed by Dr. Verkman, is focused on the identification of CF targets, the development of screening assays and screening paradigms, and the identification of drug candidates. Two Pilot Project slots will be allocated for years 16?20. Each Pilot Project runs for a maximum of 2 years, with second year funding requiring satisfactory progress and justification for continued funding. Pilot project selection involves project recruitment, letters of intent, initial review by an Internal Advisory Committee, and generally external review by our External Advisory Committee and ad hoc reviewers. Recent accomplishments of the Pilot Program include manuscripts, patents, new grants, and potential out-licensing of CF drug candidates. The proposed pilots for funding in year 16 focus on a potential new mutation-agonist CF target, SLC26A9, and therapeutic development for a rare CF-causing CFTR mutation, C.3700A>G.

Agency
National Institute of Health (NIH)
Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Type
Center Core Grants (P30)
Project #
2P30DK072517-16
Application #
10001910
Study Section
Special Emphasis Panel (ZDK1)
Project Start
2005-08-01
Project End
2023-05-31
Budget Start
2020-06-01
Budget End
2021-05-31
Support Year
16
Fiscal Year
2020
Total Cost
Indirect Cost
Name
University of California San Francisco
Department
Type
DUNS #
094878337
City
San Francisco
State
CA
Country
United States
Zip Code
94118
Smith, Alex J; Verkman, Alan S (2018) The ""glymphatic"" mechanism for solute clearance in Alzheimer's disease: game changer or unproven speculation? FASEB J 32:543-551
Duan, Tianjiao; Smith, Alex J; Verkman, Alan S (2018) Complement-dependent bystander injury to neurons in AQP4-IgG seropositive neuromyelitis optica. J Neuroinflammation 15:294
Lee, Sujin; Cil, Onur; Diez-Cecilia, Elena et al. (2018) Nanomolar-Potency 1,2,4-Triazoloquinoxaline Inhibitors of the Kidney Urea Transporter UT-A1. J Med Chem 61:3209-3217
Tradtrantip, Lukmanee; Felix, Christian M; Spirig, Rolf et al. (2018) Recombinant IgG1 Fc hexamers block cytotoxicity and pathological changes in experimental in vitro and rat models of neuromyelitis optica. Neuropharmacology 133:345-353
Phuan, Puay-Wah; Veit, Guido; Tan, Joseph-Anthony et al. (2018) ?F508-CFTR Modulator Screen Based on Cell Surface Targeting of a Chimeric Nucleotide Binding Domain 1 Reporter. SLAS Discov 23:823-831
Verkman, Alan S; Yao, Xiaoming; Smith, Alex J (2018) The evolving mystery of why skeletal muscle is spared in seropositive neuromyelitis optica. J Cell Mol Med 22:2039-2040
Sun, Dingyuan I; Tasca, Alexia; Haas, Maximilian et al. (2018) Na+/H+ Exchangers Are Required for the Development and Function of Vertebrate Mucociliary Epithelia. Cells Tissues Organs :1-14
Bhakta, Nirav R; Christenson, Stephanie A; Nerella, Srilaxmi et al. (2018) IFN-stimulated Gene Expression, Type 2 Inflammation, and Endoplasmic Reticulum Stress in Asthma. Am J Respir Crit Care Med 197:313-324
Verkman, Alan S; Smith, Alex J; Phuan, Puay-Wah et al. (2017) The aquaporin-4 water channel as a potential drug target in neurological disorders. Expert Opin Ther Targets 21:1161-1170
Zhu, Jie S; Son, Jung-Ho; Teuthorn, Andrew P et al. (2017) Diverting Reactive Intermediates Toward Unusual Chemistry: Unexpected Anthranil Products from Davis-Beirut Reaction. J Org Chem 82:10875-10882

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