HDAC inhibition protects degenerating cone photoreceptors in vivo

01/09/2015 to 29/02/2016

Hereditary cone degenerations, such as in Stargardt and Best disease, achromatopsia and cone dystrophies are caused by mutations in single genes, and lead to severe visual impairment, reduced visual acuity, and loss of color vision. We have previously shown that in an animal model for cone photoreceptor degeneration (cpfl1 mouse) follows a non-apoptotic cell death mechanism characterized by increased HDAC activity at the peak of degeneration (Arango-Gonzalez et al., PLoS One, 9:e112142, 2014). We will study the protective effects of HDAC inhibition to identify and define therapeutic window of opportunity. The use of cpfl1 animals should facilitate clinical translation to human diseases affecting cone survival and cone migration, such as cone dystrophies, age-related macular degeneration and diabetic retinopathy.