Drugs developed by Einstein’s Ana Maria Cuervo, M.D., Ph.D., and Evripidis Gavathiotis, Ph.D., show promise for treating vision in retinitis pigmentosa (RP), an inherited eye disease that can lead to blindness and is currently incurable. The research, involving a mouse model of RP and published in July 2022 in Nature Communications, is described in “Drugs Show Promise Against Eye Disease.”
In a key experiment, RP mice were intravitreally injected with the drug CA77 or a control liquid, at a time (18 days of age) when rods and cones (the retina’s photoreceptor cells) were dying. These retinal cross sections, made one week after injection, show the all-important outer segments of the rods (magenta) and cones (green), which convert light energy into electrical signals that enable vision. Beneath the outer segments are the rod and cone nuclei (blue). Compared with the retina of the control-injected mouse, the retina of the drug-injected mouse is clearly healthier, with many more photoreceptors preserved.