(click figure to enlarge) Gene therapy rescue of vision in retinal degeneration. (A) In the healthy retina, light penetrates from inner to outer retina to reach the cones and rods, which transduce signals through horizontal, bipolar, amacrine, and ultimately retinal ganglion cells to the brain. (B) In outer retinal degenerative diseases, loss of photoreceptors renders the retina insensitive to light. (C) Gene therapy with AAV2/2 virus expressing human rhodopsin (hRod) under the control of the CAG promoter results in expression of the photopigment in many surviving cells of the inner retina, and results in restoration of light responses recognized by the brain. (D) More selective expression of rhodopsin in a subset of bipolar cells is achieved by use of a virus in which expression is driven by the grm6 promoter. This version appeared to restore the most natural visual function to blind mice.
Thursday, October 15, 2015
Rhodopsin curing blindness?
In a previous life (1962-1998) my laboratory studied how the rhodopsin visual pigment in our eyes changes light into a nerve signal. Thus it excites me when I see major advances in understanding our vision and curing visual diseases. I want to pass on a nice graphic offered by Van Gelder and Kaur to illustrate recent work of Cehajic-Kapetanovic et al. (open access) showing that introduction of the visual pigment rhodopsin by viral gene therapy into the inner retina nerve cells of retinas whose rods and cones have degenerated can restore light sensitivity and can restore vision-like physiology and behavior to mice blind from outer retinal degeneration: