Regenerative Therapies for Glaucoma and Neurotrauma

Article: Cell-replacement Therapies for Visual System Disorders
Source: Vanderbilt University Medical Center, via NEI
Published: July 30, 2021

Neurons (green) and their supporting astrocytes (violet),
created in a petri dish from stem cells

Researchers will begin two translational studies aimed at restoring vision by regenerating the photoreceptors and retinal ganglion cells (RGCs) of the retina. Vision loss through death of RGCs is irreversible; however, cell replacement therapy could offer a potential solution for vision restoration. The projects will span five years and will involve labs at multiple institutions. Some of the researchers will establish and validate a squirrel monkey model of glaucoma. One of the authors explains, “We hope to develop systems that are closer to human visual anatomy, function and disease than current models...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.” Others will study models of glaucoma and neurotrauma in tree shrews, which they say have features that are relevant to humans. According to the researchers, studying both models of glaucoma and neurotrauma will provide more nuanced information, since retinal environments and cell survivability may differ between the two conditions. For example, glaucoma, being a more slowly progressing disease than neurotrauma, has a wider window in which transplantation of stem cells may be effective. Some of the researchers will focus on developing and characterizing pluripotent stem cells that can be converted into either eye organoids or directly to RGCs, and others will quantify changes in cytokine levels, glial reactivity, axon degeneration and RGC death over time. The project aims to tie the various studies together in the context of studying RGC transplant: differentiation, migration, local integration and synapse formation, growth down the optic nerve, and targeting to distal brain nuclei. The investigators from the various institutions plan to meet several times a year to encourage collaboration.

My rating of this study: ⭐⭐

"Retinal Ganglion Cell Replacement in Optic Neuropathies." NIH Reporter. 5U24EY029903-03.