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UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) - In Vitro Bioassay and Model Development Resource

UAB 儿童囊性肾病核心中心 (UAB-CCKDCC) - 体外生物测定和模型开发资源

基本信息

批准号：
10218163
负责人：
John M Parant
金额：
$ 14.69万
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/10218163
关键词：
Address Adolescent Adult Affect Alleles Bardet-Biedl Syndrome Biological Assay Biosensor CRISPR/Cas technology Cell Line Cells Childhood Cilia Communities Consultations Cyclic AMP Cyst Cystic Kidney Diseases Cystic kidney Data Defect Disease Disease model Drug Screening Engineering Epithelial Fee-for-Service Plans Fingerprint Functional disorder Generations Genes Genetic Genetic Engineering Genetic Fingerprintings Genetic Predisposition to Disease Genome Goals Human In Vitro Individual Joubert syndrome Karyotype Kidney Kidney Diseases Knock-out Laboratories Learning Maintenance Measures Meckel-Gruber syndrome Modeling Molecular Mus Mutation Nephronophthisis Organoids Outcome Pathogenesis Pathway interactions Patients Phase Proteins Quality Control Rattus Reagent Reporter Reproducibility Research Resource Development Resources Signal Pathway Site System Testing Therapeutic Urine Validation Visualization base conditional knockout embryonic stem cell experimental study fighting genetically modified cells genome editing improved in vitro Bioassay in vitro Model in vivo in vivo Model innovation member model development personalized medicine precision drugs prevent protein transport reagent standardization repaired restoration screening stable cell line tool vector

项目摘要

ABSTRACT (CORE B) Childhood Cystic Kidney Diseases (CCKDs) are debilitating disorders for which there are limited treatments available. The Holy Grail to curing CCKD is to define signaling pathways essential for cyst initiation and subsequent maintenance that can be successfully targeted with therapeutics. In vitro models of CCKD provide a rapid research tool to analyze changes in pathways, to facilitate cellular and protein visualization, are ideal starting points for discovery or validation of hypotheses and are essential for initial testing of potential treatments. Recent advancements in organoid cultures provide an attractive pre-vivo transitional approach to understand CCKD and for second phase testing of potential treatments. Despite the importance of in vitro models for CCKD research there are critical barriers preventing their efficient and effective use: 1) readily available and sharable cell based CCKD resources; 2) careful standardization of reagents that provide rigor and reproducibility across laboratories; and 3) a resource center that facilitates generation of innovative and essential cell based resources for the CCKD research community, without burdening individual labs with the cumbersome learning curve of genome editing, biosensor generation, and quality control assessment. Core B will address these barriers in the following aims: Aim 1 - To Establish In Vitro Biosensors to Study Signaling Pathways Involved in Childhood Cystic Kidney Disorders; Aim 2 – To Genetically Engineer In Vitro Models for CCKD Research; and Aim 3- To Establish and Distribute Critical In Vitro Resources for CCKD Research. Core B acts as an essential bridge between the patient derived studies in the consortium (such as UAB Core A), and the in vivo models generated by the consortium (such as UAB Core C), and therapeutic screening (such as in UAB Core D). Having a centralized CCKD in vitro biosensor and modeling resource will facilitate and enhance research in the greater PKD community and is an essential component of fighting this debilitating disease.

摘要（核心b）