This project aims to advance vision restoration using exogenous RGC transplantation in NF1-OPG mice by 1) generating and then using an immuno-incompetent strain of the NF-1 mice as recipients; 2) initiate studies aimed at increasing retinal integration of the grafted retinal ganglion cell precursors by enzymatically disrupting the barriers on the inner surface of the retina (studies will be in mice and human retinal explants); 3) produce porcine retinal ganglion cell precursors and establish equivalency to human retinal ganglion cell precursors; 4) provide cells and support to the Goldberg group for transplantation studies in NF1 pig recipients.
Investigators
Michael Young, PhD
Massachusetts Eye and Ear Infirmary
Michael Young, PhD
Massachusetts Eye and Ear Infirmary
Current Stage
Discovery
In Vivo Proof of Concept
IND Enabling
Clinical Trial Phase 1
Clinical Trial Phase 2
Clinical Trial Phase 3