Axon Regeneration and Vision Restoration in NF-1 OPG

In optic glioma (OPG), including that associated with neurofibromatosis type 1 (NF1), retinal ganglion cells (RGCs) sustain axon injury and patients lose vision, often in early life. Injury to RGC axons in the optic nerve results in an irreversible loss of vision due to the inability of RGCs to remain viable and to regenerate their axons. Seminal studies in rodents over the past 2 decades have identified strategies that allow many RGCs to survive long-term after optic nerve injury and enable some RGCs to regenerate axons from the eye to the brain. Most of these data were developed in acute trauma models of optic nerve crush, which may or may not be directly translatable to the axon damage and optic nerve environment in NF1 OPG.

In light of these promising results, we believe that the time is right for a collaborative group of investigators to join forces to achieve unprecedented levels of regeneration and to translate these findings into proof-of-concept in NF1 OPG models as well as a larger animal model, as an essential step towards eventual treatment of NF1 OPG patients.