Human Retinal Regeneration to Cure Glaucoma

Since the human eye doesn’t naturally make new neurons after injury, we aim to stimulate new neuron formation as a potential treatment for glaucoma and other optic neuropathies.

Retinal ganglion cell neurons connect the eye to the brain and when they die, whether through glaucoma or through acute trauma, this leads to permanent vision loss and blindness. Similar to how this occurs throughout the animal kingdom, we propose to develop the capability to do this in human retinal tissues through the conversion of existing Müller cells in the eye.

Retinal repair naturally occurs in many lower vertebrate species, but it hasn’t been well established in humans due to a lack of cellular and molecular tools to control cell death and track new cell formation in real time. Our proposal addresses this critical bottleneck by offering new stem cell lines engineered with built-in cell ablation and lineage reporter tools. This provides a new platform to test and validate strategies aimed at stimulating new retinal ganglion cell formation.

We anticipate that upon completion, we will have a better understanding of the mechanisms that control human retinal regeneration. Our model will provide a means to validate clinically relevant treatments aimed at restoring retinal ganglion cells that have died, such as those affected in glaucoma. This model will be made available to other researchers who hope to test therapies for regenerating the human eye after a wide range of retinal degenerations.