UC San Diego

While some vertebrate species such as zebrafish have demonstrated a robust repair and regeneration ability, the extent of this regenerative capacity in mammals is limited. In humans, it is unknown whether this process even occurs. Vision loss resulting from retinal ganglion cell (RGC) death is a leading cause of irreversible blindness. There are several areas of active research aimed at restoring retinal ganglion cells. One of them is endogenous repair. However, for this approach to work, we need to gain a better control of the developmental mechanisms leading to RGC formation. In this study, we use ectopic overexpression of four specific transcription factors involved in ganglion cell specification and differentiation to bring about the direct conversion of ganglion cells from human pluripotent stem cells. If these newly differentiated neurons turn out to be truly bonafide ganglion cells, then the idea of generating new ganglion cells to improve the prognosis of retinal diseases will be better understood.