# Xenopus as a Model System for Hydrocephaly and Ependymal Ciliogenesis

> **NIH NIH R21** · YALE UNIVERSITY · 2021 · $196,813

## Abstract

Project Summary
Congenital Hydrocephalus (CH), the pathological expansion of the cerebral ventricles due to cerebrospinal
fluid (CSF) accumulation, is a common birth defect (1 in every 1000 births) with high mortality and morbidity. In
fact, CH is the most common disease treated by pediatric neurosurgeons and despite advances in surgical
technique, palliation in many patients remains inadequate. In part, these challenging cases may be due to our
incomplete understanding of hydrocephalus pathogenesis. While advances in genomics are beginning to
identify candidate genes that may cause CH, we lack genetically tractable animal models where pathogenesis
can be analyzed. In our recent work, we paired optical coherence tomography imaging (OCT) with the frog
Xenopus tropicalis to model human congenital hydrocephalus. We demonstrated that OCT imaging of mutant
tadpoles can readily detect hallmarks of human hydrocephalus including aquaductal stenosis and/or
ventriculomegaly. In this proposal, we seek to further advance this model and develop Xenopus as a model
system that can evaluate CH candidate genes and distinguish ciliary vs. non-ciliary pathogenesis mechanisms.
Our central hypothesis is that the pathogenesis of CH due to ciliary vs. non-ciliary mechanisms will be
discretely different with respect to ventricular morphology, CSF flow network, and neural progenitor cell fate,
which can have important implications for treatment.

## Key facts

- **NIH application ID:** 10127716
- **Project number:** 5R21NS116484-02
- **Recipient organization:** YALE UNIVERSITY
- **Principal Investigator:** ENGIN DENIZ
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $196,813
- **Award type:** 5
- **Project period:** 2020-04-01 → 2022-09-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10127716

## Citation

> US National Institutes of Health, RePORTER application 10127716, Xenopus as a Model System for Hydrocephaly and Ependymal Ciliogenesis (5R21NS116484-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10127716. Licensed CC0.

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