Viral Mediated Therapy for Photoreceptor Disease

The mammalian retina is an ideally suited target organ for developing gene-based therapies for several key reasons. First, the cell types primarily affected in known retinal diseases, retinal ganglion cells on the inner face of the retina, photoreceptors on its outer surface and retinal pigmented epithelial cells adjacent to and interdigitated with photoreceptors, are all readily accessible to the skilled surgeon.Second, there is perhaps a deeper appreciation of the range of genetic diseases of the retina than for any other organ, viz. there are currently about 150 known genetic retinal disease loci in the human genome, of which approximately 90 are known at the complete sequence level.Third, noninvasive assessment of retinal function and structure has been the focus of clinical ophthalmology for more than a century.