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UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) - Therapeutic Development and Screening Resource

UAB 儿童囊性肾病核心中心 (UAB-CCKDCC) - 治疗开发和筛查资源

基本信息

批准号：
10218165
负责人：
Michal Mrug
金额：
$ 14.06万
依托单位：
UNIVERSITY OF ALABAMA AT BIRMINGHAM
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-07-20 至 2025-06-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10218165
关键词：
Address Advanced Development Affect Americas Animal Disease Models Animal Model Autosomal Dominant Polycystic Kidney Biocompatible Materials Biological Biological Assay Biological Models Biosensor Cell Line Cells Childhood Cilia Clinical Clinical Data Clinical Trials Collaborations Complex Consultations Consumption Cyclic AMP Cyst Cystic Kidney Diseases Data Development Development Plans Disease Disease Outcome Disease Progression Disease model Engineering Equipment FDA approved Family Future Gene Mutation Genes Genetic Models Goals Hereditary Disease Human Image Impairment In Vitro Individual Intervention Intuition Laboratories Lead Medical Genetics Metabolic Methodology Mission Modeling Molecular Morphology Mutation National Institute of Diabetes and Digestive and Kidney Diseases Nephronophthisis Observational Study Outcome Pathway interactions Patient-Focused Outcomes Patients Pharmaceutical Preparations Preclinical Testing Recruitment Activity Renal function Reporter Reproducibility Research Research Personnel Resource Development Resources Scientist Services Signal Pathway Signal Transduction Site Societies Specimen Standardization System Techniques Therapeutic Therapeutic Uses Time Toxic effect Toxicology Translations Vision Work base biobank clinical care cohort cost cost effective design effectiveness evaluation effectiveness testing efficacy evaluation improved improved outcome in vitro Bioassay in vivo in vivo Bioassay in vivo Model in vivo evaluation induced pluripotent stem cell kidney cell loss of function member model development multidisciplinary novel therapeutics patient population pre-clinical prevent protein function recruit screening sensor serial imaging therapeutic candidate therapeutic development therapy development tool translational study treatment strategy

项目摘要

ABSTRACT (CORE D) It is the goal of the UAB-Childhood Cystic Kidney Disease Core Center (CCKDCC) to work with the other U54 PKD Centers (RTCC), under the direction of the U24 Coordinating Center Site (U24-CCS) to reduce obstacles in cystic kidney disease research leading to more rapid translational studies by PKD Consortium Members and ultimately to the development of novel therapeutics. The UAB-CCKDCC has assembled a multidisciplinary team of researchers to carry out these goals and actively recruit new and established investigators to the field. The Center will help address the limitations associated with the small CCKD patient population available at individual intuitions by providing access to clinical data and biomaterial from human CCKD patients from across the Americas. The Center will focus on the development of resources to analyze cilio-cystic disease protein function, localization, and interactions. The Center will generate and provide researchers with patient relevant models of CCKD and establish methodology to utilize these models to ascertain the efficacy of candidate therapies to slow disease progression using a standardized, cost-effective, and longitudinal imaging strategy. The resources provided by the CCKDCC will help address several of the most significant hurdles slowing the development of therapeutic approaches and strategically recruit new scientists into the field. The Therapeutics Development and Screening Core (Core D) will coordinate efforts with other U54-PKD Centers, under the direction of the U24-CCS and NIDDK to become a shared national resource facility in which a PKD Consortium member can rapidly evaluate the ability of a candidate therapeutic drug to reverse alterations in pathways commonly associated with CCKD gene mutations and to prevent cyst initiation and expansion and renal function loss. This centralized resource is designed to accelerate discovery research in CCKDs in partnership with the UAB-CCKDCC In vitro and In vivo Bioassay and Model Development Resources (Cores B and C). Integration of these resources with clinical and genetic data and biological specimens from patients with CCKDs, as well as the US node of ADPedKD (a multicenter observational study of childhood ADPKD) compiled by the Clinical, Translational, and Biorepository Resource (Core A) will directly inform the development of new, targeted interventional strategies for patients with CCKDs.

摘要（核心d）