Combined stem cell and trophic factor therapy for glaucoma

Project Summary

AIM 1: Compare retinal ganglion cell protection following delivery of hNPC-GDNF close to the optic nerve head and intravitreaou delivery of hNPC-GDNF. AIM 2: Dose range study to find out whether retinal ganglion cell protection after delivery of hNPC-GDNF is dose-dependent. AIM3: Mechanism of hNPC-GDNF in protecting retinal ganglion cells by study gene expression of astrocytes (at the optic nerve head), retinal ganglion cells and hNPC-GDNF 

Delivering combined neural progenitor cells and trophic factor close to the optic nerve head-site of the glaucoma for retinal ganglion cell protection. Using a well-characterized cell and gene product that has shown neuroprotection in multiple animal model and demonstrated safety profile in recently completed clinical trial for ALS. 

If delivery of hNPC-GDNF close to the optic nerve head (site of the disease) is more effective than intravitreous injection, this study can be potentially translated into clinic for treating glaucoma. The findings from AIM 3 study will identify top regulated genes that play critical role in retinal ganglion cell protection and aid to develop specific targeted molecular therapy for glaucoma.