# Embryonic Stem Cell Approach to Retinal Ganglion Cell Replacement: an In Vivo Study

> **NIH NIH R01** · UNIVERSITY OF CALIFORNIA AT DAVIS · 2020 · $392,500

## Abstract

Project Summary
Several human pathologies lead to retinal ganglion cell (RGC) degeneration and irreversible vision loss.
Stem cell-based cell replacement therapy offers promising novel approaches for vision restoration. However,
there are many issues to overcome before these therapies can be clinically trialled: The current methods to
differentiate embryonic stem cells (ESCs) into retinal fates yield modest numbers of RGCs, and previous
reports have suggested that only a small fraction of RGCs can integrate with a host retina upon
transplantation. The main goals of the present proposal are: (1) to improve the current protocols to efficiently
differentiate ESCs into RGC fates at the optimal age for transplantation, and (2) to identify possible barriers
to cell engraftment.
1) To generate high yields of ESC-derived RGCs: Our lab and others have implemented methods to
differentiate embryonic stem cells into bona-fide retinal progenitors. We will develop the current protocols to
increase the production of RGCs by systematically testing an array signaling cues and miRNA regulators
known to be involved in RGC differentiation during normal development. By using a variety of approaches
including RNA-sequencing and electrophysiology, we will characterize the ESC-derived RGCs. We will label,
purify and transplant RGCs from an array of different ages to pinpoint the optimal developmental stage to
become donor cells for transplantation.
2) To analyze integration potential of ESC-derived RGCs following transplantation: We will use state-
of-the-art imaging technologies to follow individual ESC-derived RGCs after transplantation in living rodents
to accurately measure survival, migration and integration with the host. We will employ murine models of
RGC degeneration to assay the role of the environment in RGC engraftment potential.

## Key facts

- **NIH application ID:** 9932989
- **Project number:** 5R01EY026942-05
- **Recipient organization:** UNIVERSITY OF CALIFORNIA AT DAVIS
- **Principal Investigator:** Anna La Torre
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $392,500
- **Award type:** 5
- **Project period:** 2016-08-01 → 2022-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9932989

## Citation

> US National Institutes of Health, RePORTER application 9932989, Embryonic Stem Cell Approach to Retinal Ganglion Cell Replacement: an In Vivo Study (5R01EY026942-05). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9932989. Licensed CC0.

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