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

> **NIH NIH U54** · UNIVERSITY OF ALABAMA AT BIRMINGHAM · 2020 · $143,288

## Abstract

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.

## Key facts

- **NIH application ID:** 10058130
- **Project number:** 1U54DK126087-01
- **Recipient organization:** UNIVERSITY OF ALABAMA AT BIRMINGHAM
- **Principal Investigator:** Michal Mrug
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $143,288
- **Award type:** 1
- **Project period:** — → —

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10058130

## Citation

> US National Institutes of Health, RePORTER application 10058130, UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) - Therapeutic Development and Screening Resource (1U54DK126087-01). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10058130. Licensed CC0.

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