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.
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.
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