The Hu Lab at Stanford has developed a neuroprotective gene therapy for treating glaucoma and other optic neuropathies. Their gene therapy AAV vector expresses NMNAT2 operably linked to a retinal ganglion cell-specific promoter (mSngc).
Stanford researchers have developed a gene therapy that combines a retinal ganglion cell (RGC)- specific promoter with CRISPR gene editing to provide effective neuroprotection in optic neuropathies.