New strategies of protecting RGCs and promoting optic nerve regeneration

Vision loss associated with some patients of neurofibromatosis type 1 (NF1) is characterized by degeneration of the optic nerve and the loss of retinal ganglion cells (RGCs), following optic pathway glioma (OPG). Currently there is no solution to reverse the optic pathway damage and restore vision. Tremendous progress in both NF1/OPG and RGC biology provide an unprecedented opportunity of tacking this issue and developing relevant therapeutic strategies.

The objective of this project is to leverage our expertise in studying RGC survival and regeneration to explore novel neuroprotective and restorative approaches for NF1-OPG. As to protective therapies, we will assess the effects of available axon-protecting manipulations in NF1-OPG and also identify novel protective interventions targeting the vulnerability of RGCs of NF1-OPG. In addition, we will collaborate with exogenous cell replacement groups in developing a set of manipulations that maximize the extents of axon growth, so that transplanted RGCs could reach their targets for vision restoration. As a component of VRI team, these proposed studies are expected to contribute to the development of vision restoration therapies for NF1-OPG patients.

Investigators

Zhigang He, PhD

Boston Children's Hospital

Zhigang He, PhD

Boston Children's Hospital

Current Stage

Discovery

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