The Hepatorenal Fibrocystic Disease Core Center (HRFDCC) is an established Center with a unique focus on Autosomal Recessive Polycystic Kidney Disease (ARPKD) and other single gene recessive HRFD disorders. The Center's Cores are multi-institutional providing unique opportunities for integrating the research and clinical expertise across the institutions and for leveraging institutional support. Further, the HRFDCC coordinates with other NIH and University supported Centers including the UAB/UCSD O'Brien Center, UAB Nephrology Research and Training Center, and the Clinical and the Translational Science Award (CTSA) programs to expand activities far beyond that possible with only the P30 support. The overarching goals of the HRFDCC are: 1) to attract and support innovative research into the causes of HRFDs; 2) to facilitate the use of scientifically rigorous, state-of-th-art methodologies in a cost-effective manner to address HRFD pathogenesis; 3) to expand capabilities and specificity of HRFD diagnostic tools; and 4) to promote the development of efficacious treatments for HRFDs. The Center supports a large national and international Investigator Base, each with a rigorous research program in one or more of the Center's thematic areas of interest that include: 1) cilia biology; 2) epithelial transport processes; 3) regulation of epithelial proliferation, apoptosis, and differentiation; 4) epithelial injury and reair mechanisms; 5) drug discovery and treatment strategies; and 6) improved approaches for HRFD diagnostics. The Center supports and enhances the research activities of the members through four biomedical Cores that include: 1) The HRFDC Translational Resource (Core A); 2) The Engineered Models Resource (Core B); 3) The Cellular Physiology Resource (Core C); and 4) The Therapeutics Screening and Drug Development Resource (Core D). Importantly, all of these Cores are integrated such that new resources and methodologies established by one Core expand opportunities and services offered by the other Cores. In addition, mentored Pilot and Feasibility and Voucher Programs are in place to support the development of meritorious projects. The Center also provides an Educational Enrichment Program consisting of a seminar series, an annual scientific symposium, workshops, renal chalk talks, and training and mentoring sessions. These activities support junior faculty/fellows/students and attract new investigators into the HRFD field while facilitating strong interactions among the Investigator Base. Collectively, the Center's Cores, with their associated cohort of interdisciplinary investigators, provide the breadth and depth of expertise that is crucial for innovative and productive advances in HRFD research. The Center offers significant added value for investigators through attracting institutional commitments, through pilot project support, by prompting interactions between basic and clinical scientists, and through its Core services and development of innovative resources. Cooperatively, the HRFDCC will expedite the translation of basic discoveries into better diagnosis and treatment strategies for HRFDs.

Public Health Relevance

Hepatorenal fibrocystic diseases (HRFDs), such as ARPKD, are devastating disorders causing significant childhood morbidity and mortality. Remarkable progress has been made in identifying genetic factors responsible for HRFDs. However, our knowledge about how these genetic defects lead to HRFDs is still limited, hindering the development of effective therapeutic strategies. The mission of the Hepatorenal Fibrocystic Disease Core Center (HRFDCC) is to accelerate the pace of research into the genetic, biochemical, and cellular mechanisms of HRFDs and the subsequent translation of these discoveries into effective treatments. To advance HRFD research, the HRFDCC will integrate the expertise of the Core Directors and the Investigator Base with genetic information from HRFD patients, cost effective methodologies, and access to innovative in vitro and in vivo HRFD biological resources. The Center will complement this integrative model with infrastructure allowing efficient preclinical assessment of lead compounds across multiple HRFD diseases using cell-based and in vivo models. Together, the activities of the Center will promote more rapid advancement of efficacious candidates into clinical trials for human HRFD patients.

Agency
National Institute of Health (NIH)
Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Type
Center Core Grants (P30)
Project #
3P30DK074038-11S1
Application #
9264673
Study Section
Special Emphasis Panel (ZDK1)
Program Officer
Flessner, Michael Francis
Project Start
2005-09-30
Project End
2020-06-30
Budget Start
2015-09-25
Budget End
2016-06-30
Support Year
11
Fiscal Year
2016
Total Cost
Indirect Cost
Name
University of Alabama Birmingham
Department
Anatomy/Cell Biology
Type
Schools of Medicine
DUNS #
063690705
City
Birmingham
State
AL
Country
United States
Zip Code
35294
Lobo, Glenn P; Pauer, Gayle; Lipschutz, Joshua H et al. (2018) The Retinol-Binding Protein Receptor 2 (Rbpr2) Is Required for Photoreceptor Survival and Visual Function in the Zebrafish. Adv Exp Med Biol 1074:569-576
Bevensee, Mark O (2018) A new coupling of an acid-base transporter to PKD and cyst formation. J Physiol :
Bignall 2nd, O N Ray; Dixon, Bradley P (2018) Management of Hematuria in Children. Curr Treat Options Pediatr 4:333-349
Engle, Staci E; Antonellis, Patrick J; Whitehouse, Logan S et al. (2018) A CreER mouse to study melanin concentrating hormone signaling in the developing brain. Genesis 56:e23217
Desai, Paurav B; San Agustin, Jovenal T; Stuck, Michael W et al. (2018) Ift25 is not a cystic kidney disease gene but is required for early steps of kidney development. Mech Dev 151:10-17
Vuong, Linh T; Iomini, Carlo; Balmer, Sophie et al. (2018) Kinesin-2 and IFT-A act as a complex promoting nuclear localization of ?-catenin during Wnt signalling. Nat Commun 9:5304
Jo, SeongHo; Chen, Junqin; Xu, Guanlan et al. (2018) miR-204 Controls Glucagon-Like Peptide 1 Receptor Expression and Agonist Function. Diabetes 67:256-264
Polgar, Noemi; Fogelgren, Ben (2018) Regulation of Cell Polarity by Exocyst-Mediated Trafficking. Cold Spring Harb Perspect Biol 10:
Chumley, Phillip; Zhou, Juling; Mrug, Sylvie et al. (2018) Truncating PKHD1 and PKD2 mutations alter energy metabolism. Am J Physiol Renal Physiol :
Jiang, Lu; Fang, Pingping; Septer, Seth et al. (2018) Inhibition of Mast Cell Degranulation With Cromolyn Sodium Exhibits Organ-Specific Effects in Polycystic Kidney (PCK) Rats. Int J Toxicol 37:308-326

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