# UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC)

> **NIH NIH U54** · UNIVERSITY OF ALABAMA AT BIRMINGHAM · 2023 · $180,448

## Abstract

SUPPLEMENTAL REQUEST ABSTRACT
 The overall objective of the In Vivo Models Resource (IVMR) in the UAB Childhood Cystic Kidney Disease
Center (CCKDC) is to enable the research community to study mechanisms driving renal cystogenesis and to
facilitate the translation of basic discoveries into clinical care for PKD patients. As indicated in the specific aims
from the original application, we are accomplishing these goals through the generation and distribution of mice
with PKD/ciliopathy mutations, PKD protein reporter lines, and biosensors for pathways associated with cystic
kidney disorders. However, there is an additional demand for PKD patient relevant rat models based on multiple
resource requests and feedback we've received from PKD investigators that participated in the PKD RRC Annual
Symposium and the FASEB PKD Summer Research Conference. The generation of rat PKD models was not
included in the scope of work or budget of the original proposal. Thus, the purpose of this supplement is to
request funds that will allow the UAB IVMR to generate patient variant models for PKD1 and PKD2, along with
conditional flox alleles. In the absence of this supplemental support, we would not be able to generate these
models to fulfil the resource request from the research community.
 Null mutations in PKD1 and PKD2 in rats are lethal. Thus, we are proposing to generate alleles that were
identified as homozygous changes in human ADPKD patients and thus are likely hypomorphic mutations. The
PKD1(R3268C) and PKD2(L656W) models we are generating will be the first patient relevant lines to be
established in the rat system. The floxed alleles will facilitate conditional deletion for temporal analysis of gene
function and cyst formation and would be utilized in genetic crosses with the PKD1(R3268C) and PKD2(L656W)
in the off chance that the hypomorphic alleles are homozygous lethal. All the new rat lines we generate will be
distributed through the PKD RRC as soon as they pass quality control standards. Collectively these new models
will provide expanded opportunities for preclinical drug testing, analysis of disease mechanisms, and will facilitate
renal physiology studies that are exceedingly challenging using the mouse models.

## Key facts

- **NIH application ID:** 10892542
- **Project number:** 3U54DK126087-04S1
- **Recipient organization:** UNIVERSITY OF ALABAMA AT BIRMINGHAM
- **Principal Investigator:** Bradley K. Yoder
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $180,448
- **Award type:** 3
- **Project period:** 2020-07-20 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10892542

## Citation

> US National Institutes of Health, RePORTER application 10892542, UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) (3U54DK126087-04S1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10892542. Licensed CC0.

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