# Development of small-molecule Wnt mimetics for corneal epithelial cell regeneration

> **NIH NIH R01** · UNIVERSITY OF CALIFORNIA LOS ANGELES · 2020 · $497,177

## Abstract

Abstract
Limbal stem cell deficiency (LSCD) is a major cause, either primary or secondary, of significant visual loss and
blindness in many common corneal disorders. Transplantation of autologous limbal stem cells (LSCs)
expanded in tissue culture has successfully restored vision and revolutionized the treatment of LSCD. A higher
expansion efficiency of the stem/progenitor cell population in culture corresponds to a greater probability of
long-term graft survival. The long-term goal of our study is to develop small molecules that can govern LSC
self-renewal and differentiation and be used as therapeutic reagents for stem cell-based treatments of LSCD–
related disorders. The most efficient expansion method requires feeder cells that provide a proper
microenvironment to support the growth of LSCs. Among the external signaling that the feeder cells provide to
the LSCs is the Wnt signaling. The central hypothesis of this project is that small molecules can be developed
to mimic the Wnt proteins and activate Wnt signaling in the cells. We further propose that these small
molecules will increase the efficiency of ex vivo expansion of functional human LSCs. The goal of this
application is to use a structure-based drug discovery approach to develop potent Wnt mimics small molecule
and to test their ability to increase the efficiency of ex vivo LSC expansion. Because the maintenance of stem
cell characteristics in the process of culture expansion is essential for the success of ocular surface
reconstruction, the small molecules generated in this project will serve as a platform for the development of
novel pharmaceutical reagents for treating other corneal epithelial disorders.

## Key facts

- **NIH application ID:** 10000159
- **Project number:** 5R01EY028557-03
- **Recipient organization:** UNIVERSITY OF CALIFORNIA LOS ANGELES
- **Principal Investigator:** Sophie Deng
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $497,177
- **Award type:** 5
- **Project period:** 2018-09-30 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10000159

## Citation

> US National Institutes of Health, RePORTER application 10000159, Development of small-molecule Wnt mimetics for corneal epithelial cell regeneration (5R01EY028557-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10000159. Licensed CC0.

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