# Retinal Ganglion Cell Replacement in Optic Neuropathies

> **NIH NIH U24** · STANFORD UNIVERSITY · 2021 · $1,357,464

## Abstract

PROJECT SUMMARY
 Glaucoma is a leading causes of blindness and along with other optic neuropathies is characterized by
the loss of retinal ganglion cells (RGCs). Increased intraocular pressure (IOP) management is the current
standard of care for glaucoma patients, but fails to stop the irreversible loss of RGCs and progressive visual
dysfunction. Vision restoration through RGC replacement therapy, one of the NEI’s Audacious Goals program,
could be a potential solution, and considerable progress has been made in understanding the molecular
signals that regulate RGC specification from human stem cells, as well as in RGC transplant and integration in
rodents. However, when considering translation of laboratory advances to human testing, rodent models are
limited by critical differences in retinal physiology, and proof-of-concept in non-human primates would greatly
increase confidence and aid in therapeutic development before moving to human testing. Thus there is a
considerable need for a tractable non-human primate model. Here we will establish a squirrel monkey-induced
glaucoma model and the parameters to study human stem cell-derived RGC integration and potential vision
restoration in a retina and visual system closer to those of human. Through this 5-year proposal we will
achieve critical milestones, including validating the monkey glaucoma model, studying key structural and
functional measures using innovative new modalities that should be portable between monkey and humans,
and demonstrating the model’s ability to move across institutions. All of this will be accomplished in the setting
of studying RGC transplant: differentiation, migration, local integration and synapse formation, growth down the
optic nerve, and targeting to distal brain nuclei, with the goal of vision restoration.

## Key facts

- **NIH application ID:** 10239017
- **Project number:** 5U24EY029903-04
- **Recipient organization:** STANFORD UNIVERSITY
- **Principal Investigator:** David J. Calkins
- **Activity code:** U24 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $1,357,464
- **Award type:** 5
- **Project period:** 2018-09-30 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10239017

## Citation

> US National Institutes of Health, RePORTER application 10239017, Retinal Ganglion Cell Replacement in Optic Neuropathies (5U24EY029903-04). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/10239017. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*