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

> **NIH NIH U54** · UNIVERSITY OF ALABAMA AT BIRMINGHAM · 2023 · $163,921

## Abstract

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.

## Key facts

- **NIH application ID:** 10685989
- **Project number:** 5U54DK126087-04
- **Recipient organization:** UNIVERSITY OF ALABAMA AT BIRMINGHAM
- **Principal Investigator:** John M Parant
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $163,921
- **Award type:** 5
- **Project period:** 2020-07-20 → 2025-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10685989

## Citation

> US National Institutes of Health, RePORTER application 10685989, UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) - In Vitro Bioassay and Model Development Resource (5U54DK126087-04). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10685989. Licensed CC0.

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