HDAC inhibition as a novel tool for cone photoreceptors neuroprotection

31/05/2019 to 31/05/2020

Mutation-independent secondary cone degeneration, as it happens in Retinitis Pigmentosa (RP), is a main contributor to inherited blindness in the working age population. RP patients refer to ophthalmologists usually only when most rods have already degenerated and cone degeneration has set in. Therefore, from a clinical view-point, stopping secondary cone death is most important and has the potential to preserve useful vision in patients suffering from RP.
The design of novel rational therapies aiming to prevent cone death requires an in-depth knowledge on the molecular processes leading to cone cell death. We have previously shown that both primary rod and primary cone degeneration are tightly regulated by aberrant HDAC activity. Our recent data suggest that epigenetic modifications are also associated with secondary cone degeneration. These findings suggest a unique possibility for targeted pharmacological protection of both primary degenerating rods and mutation-free secondary dying cones and creates hope to maintain vision in RP patients independent of the disease stage.