Pilot & Feasibility Program (P&F)
The Pilot and Feasibility program encourages young and established investigators to undertake research relevant to kidney development, engineering and disease multiomics to accelerate the acquisition of knowledge that can be applied to treatment of children with renal and urological diseases and engineer devices to replenish kidney function.
The areas supported by the PCEN-WU include kidney development, experiments using iPSCs, pediatric kidney diseases (CAKUT, glomerular diseases, AKI, CKD), tissue engineering, disease modeling, pediatric tissue-based research, multiomics studies related to single cell and spatially resolved methods to understand kidney biology. Human and non-human model system studies are eligible. Use of PCEN core services is NOT a requirement.
Awardees
| PI | Award year | Career stage | Institution | Title: | Description: |
|---|---|---|---|---|---|
| Kyle McCracken, MD, PhD | 2023 | Early | Cincinnati Children’s Hospital Medical Center | Regulation of cell fate determination in the ureteric bud lineage in hPSC-derived organoids | In this project, we will use human pluripotent stem cell-derived organoids and fluorescent lineage reporters to elucidate the mechanisms that regulate cell fate decisions in the developing urinary collecting system. These pilot studies will inform our approach and ability to generate distinct types of epithelial tissues from stem cells, including the ureter and collecting duct, which will have diverse applications in kidney tissue engineering and disease modeling. |
| Moe R. Mahjoub, PhD | 2023 | Established | Washington University in St. Louis | Characterizing Centrosomal Pathways Underlying Congenital Kidney Developmental Defects and Early-Onset Fibrocystic Disease | Congenital fibrocystic kidney “ciliopathies” comprise a group of pediatric disorders caused by mutations in genes associated with the centrosome-cilium organelle complex. There are no effective molecular therapies that successfully halt disease progression. Understanding the underlying cellular and molecular mechanisms that contribute to the disease is critical in developing an effective therapeutic strategy. The goal of this project is to identify pathways that are disrupted in the rare, early-onset pediatric renal ciliopathy diseases caused by mutations in centrosome-associated genes. These experiments will be performed using a new mouse model of pediatric ciliopathies, together with single-cell sequencing approaches. |
| Albert Liu, MD | 2023 | Early | Washington University in St. Louis | Mechanism of Hypertension in Metabolic Syndrome and Post-Transplant Obesity and Diabetes | Metabolic syndrome is a complex constellation of obesity, insulin resistance, dyslipidemia, and hypertension. In metabolic syndrome, the hypertension is thought to be due to endothelial dysfunction as a result of the other components of metabolic syndrome. Glucagon-like peptide-1 receptor agonists (GLP-1RA) are a class of medication originally approved for type 2 diabetes mellitus with recent approval for obesity, and use of these medications have been colloquially associated with decreased need for antihypertensive medications. We aim to assess the relationship of GLP-1RA use and associated need for antihypertensive medications, and also assess vascular function using non-invasive methods (pulse wave analysis, pulse wave velocity, carotid intima-media thickness, and flow-mediated dilation) to assess vascular functioning in pediatric patients with metabolic syndrome as compared to healthy individuals, as well as assess changes in these assessments in patients with metabolic syndrome receiving GLP-1RA therapy. |
News
Request for Applications for 2024 Pilot and Feasibility Program
The Pediatric Centers of Excellence in Nephrology (PCENs) at the University of Virginia (UVA), Children’s Hospital of Philadelphia (CHOP), and Washington University in St. Louis (WashU) are issuing a joint RFA for Pilot and Feasibility (P&F) projects. The PCEN program is funded by the National Institute of Diabetes and Digestive and Kidney Diseases.