There are so many different approaches on the horizon for treating inherited retinal diseases, like Retinitis Pigmentosa (PR) and Leber’s Congenital Amaurosis (LCA).
Here’s a new one that is working in mouse models and seems really promising! So you know how the retina is comprised of rods and cones? Well, apparently when there is retinal degeneration the rods tend to die off first and as the rods are lost the cones quickly follow because cones depend on rods for their survival.
This means that, logically speaking, if we can save the rods then we should also be able to prevent cone damage as well.
With this in mind, researchers decided to try a new approach targeting rods… and tricking them into thinking they’re really cones! Using a compound that blocks a certain gene activity, researchers were able to turn rods more cone-like in mouse models and preserve them from degeneration.
What is the benefit? Well, if the rods don’t die, then the cones are also preserved. However, now with the rods acting more like cones it does mean that patients would lose the vision abilities usually supported by rods (such as night and peripheral vision).
Yes, it’s a little confusing, but also pretty fascinating! You can read more about this approach on the Foundation Fighting Blindness blog.