Update from the Catalyst for a Cure Vision Restoration Initiative

The Catalyst for a Cure Vision Restoration team has identified a set of three genes that, when inhibited, improve the survival of optic nerve cells in models of glaucoma.

The Catalyst for a Cure Vision Restoration research team is exploring and developing novel strategies to protect, repair, and replace lost retinal nerve cells and help them reconnect with the visual brain.

Glaucoma is a complex disease in which damage to the optic nerve leads to progressive vision loss. Today’s treatments, which work by lowering pressure in the eye, can only preserve remaining vision. They don’t improve or restore vision that already has been lost to glaucoma.

One strategy for vision restoration in patients with advanced glaucoma is to transplant in new optic nerve cells derived from stem cells. In the past, this approach has been hampered by the poor survival of transplanted cells.

The Catalyst for a Cure Vision Restoration team has identified a set of three genes that, when inhibited, improve the survival of optic nerve cells in models of glaucoma. Recently, the team has demonstrated that this same approach (of inhibiting these three genes) can be used to dramatically improve the survival of transplanted optic nerve cells. Although preliminary, the team is continuing to investigate this promising direction.

In addition, using an exciting new screening approach in models of glaucoma, the team identified a novel set of genes that can promote optic nerve survival and regeneration. Thus, the team has now identified potentially harmful genes that need to be “turned off” and protective genes that need to be “turned on.” Our future work will be looking at whether combining these two leads might further improve optic nerve regeneration and optic nerve cell transplantation.

Moreover, for those patients who have not completely lost vision, these approaches represent unique opportunities to slow or halt the degenerative process to preserve and maintain vision.

The principal investigators in the Catalyst for a Cure Vision Restoration Initiative (pictured left to right) are Xin Duan, PhD (UC San Francisco), Yang Hu, MD, PhD (Stanford), Anna La Torre, PhD (UC Davis), and Derek Welsbie, MD, PhD (UC San Diego).

Posted December 12, 2022. Updated June 28, 2024.