Retina Regeneration in Zebrafish – Endogenous RGC replacement

NF1 is a disease resulting from reduced levels of neurofibromin 1 that functions as a tumor suppressor. One in five children with NF1 have optic gliomas that often leads to retinal ganglion cell death and blindness. Although there are many approaches one can investigate for treating vision loss in NF1 patients, we are focusing on one that takes advantage of endogenous retinal stem cells for regenerating lost retinal ganglion cells.

Müller glia are a source of multipotent retinal progenitors that regenerate retinal ganglion cells in the zebrafish retina. In this project, we plan to explore the mechanism underlying this process in zebrafish to inform us of strategies for stimulating a similar process in mammals and thus provide a therapy for people suffering from blinding eye disease, like NF1. With this goal in mind, we aim to identify gene expression changes and small molecules that are sufficient to drive retina regeneration in zebrafish and test their effect on retina regeneration in mammals.

Current Stage


In Vivo Proof of Concept
IND Enabling
Clinical Trial Phase 1
Clinical Trial Phase 2
Clinical Trial Phase 3