HDAC inhibition ameliorates cone survival in retinitis pigmentosa mice

M Samardzija, A Corna, R Gomez-Sintes… - Cell Death & …, 2021 - nature.com
M Samardzija, A Corna, R Gomez-Sintes, MA Jarboui, A Armento, JE Roger, E Petridou
Cell Death & Differentiation, 2021nature.com
Cone photoreceptor cell death in inherited retinal diseases, such as Retinitis Pigmentosa
(RP), leads to the loss of high acuity and color vision and, ultimately to blindness. In RP, a
vast number of mutations perturb the structure and function of rod photoreceptors, while
cones remain initially unaffected. Extensive rod loss in advanced stages of the disease
triggers cone death by a mechanism that is still largely unknown. Here, we show that
secondary cone cell death in animal models for RP is associated with increased activity of …
Abstract
Cone photoreceptor cell death in inherited retinal diseases, such as Retinitis Pigmentosa (RP), leads to the loss of high acuity and color vision and, ultimately to blindness. In RP, a vast number of mutations perturb the structure and function of rod photoreceptors, while cones remain initially unaffected. Extensive rod loss in advanced stages of the disease triggers cone death by a mechanism that is still largely unknown. Here, we show that secondary cone cell death in animal models for RP is associated with increased activity of histone deacetylates (HDACs). A single intravitreal injection of an HDAC inhibitor at late stages of the disease, when the majority of rods have already degenerated, was sufficient to delay cone death and support long-term cone survival in two mouse models for RP, affected by mutations in the phosphodiesterase 6b gene. Moreover, the surviving cones remained light-sensitive, leading to an improvement in visual function. RNA-seq analysis of protected cones demonstrated that HDAC inhibition initiated multi-level protection via regulation of different pro-survival pathways, including MAPK, PI3K-Akt, and autophagy. This study suggests a unique opportunity for targeted pharmacological protection of secondary dying cones by HDAC inhibition and creates hope to maintain vision in RP patients even in advanced disease stages.
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