Gene Therapy Vector for Eye Disease

Stanford researchers have discovered the first of its kind gene therapy vector to treat eye diseases of the non-pigmented ciliary epithelium cells (NPCECs). With a gene therapy vector, a more predictable and stable treatment is possible to treat glaucoma and inflammatory conditions of the eyes when utilizing specific gene targets. The Mahajan Laboratory at Stanford used tissue culture in vitro to identify a minimal DNA promotor to drive gene expression in the NPCECs. The method has shown that a BEST2 minimal promoter (specific to NPCECs gene expression) can be manipulated and linked to a sequence of gene encoding of interest in the NPCECs to treat eye disease. Primarily, NPCECs are linked to glaucoma which affects 60 million people worldwide with no cure. Current management involves delaying the progression of disease, but many people continue to lose eyesight (due to retinal ganglion cell loss) permanently even with proper therapies. NPCECs are also involved in inflammatory conditions (anterior/iridocyclitis), pseudoexfoliation syndrome, and uveal melanoma. With extended research, this technology can provide a cure and improve treatment of a vast array of eye diseases that lack effective therapy.

Stage of Development