UAB Childhood Cystic Kidney Disease Center (UAB-CCKDC)

University Of Alabama At Birmingham, Birmingham AL

Investigators

Linked publications & trials

Abstract

The Childhood Cystic Kidney Disease Center (CCKDC) is a vital component of the PKD Research Resource Consortium (PKD RRC), dedicated to advancing research in polycystic kidney disease (PKD). Our primary aim is to accelerate breakthroughs in understanding cyst development to devise effective treatments, ultimately enhancing patient care. By closely collaborating with other PKD Centers, the NIDDK, and the Steering Committee, we are committed to realizing the PKD RRC’s mission. The CCKDC’s Childhood Clinical and Translational Resource (CCTR), which has a unique focus on childhood cystic kidney disorders, will collect and integrate new patient data and biomaterials into a harmonized PKD RRC centralized database and biorepository for clinical studies. The CCTR is also developing new methodology to assess patient/caregiver experiences to better design and conduct patient-centered research activities. The Bioengineering Resource (BR) will develop cutting-edge inducible PKD mutant rat models, new in vivo and in vitro resources to detect and analyze the endogenous PKD proteins under their native conditions in live cells and tissues, and a human induced pluripotent stem cells (IPSCs) derived kidney organoid platform for rapid large-scale analysis of cystogenic pathways and high throughput functionality testing PKD proteins and patient variants. The Informatic and Data Analytics Resource (IDAR) is establishing a comprehensive hub for high-quality, large-scale omic datasets. We provide user-friendly, web-based tools designed for rigorous cross-species analyses, aiding in pathway identification and therapeutic predictions. Our consultation services enhance hypothesis generation and drug target prioritization, fostering data-driven discoveries. Moreover, the Resource Development Core (RDC) extends the consortium’s research capabilities by offering advanced genome engineering, disease modeling, and a robust organoid screening platform for pathway and drug prioritization. Through strategic collaborations with other UAB Centers and Divisions, the CCKDC is enriching the PKD research environment by recruiting top faculty, conducting summer research training programs, and organizing renal workshops and symposia. We are leveraging significant institutional support that will expand our suite of research tools, resources, and analytical programs, and enhance our educational initiatives. These efforts are amplifying the CCKDC’s overall impact on PKD research and patient care, driving transformative advancements in the field.