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Nawajes A Mandal; Therapeutic potential of targeting ceramides for the treatment of retinal diseases. Invest. Ophthalmol. Vis. Sci. 2019;60(9):4214.
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Presentation Description :
Photoreceptor cell death is the ultimate irreversible event in many forms of retinal degenerative diseases including Retinitis Pigmentosa (RP) and age-related macular degeneration (AMD). Currently, there is no therapy that can effectively blocks the progression of retinal degeneration and certainly none that can restore vision or reverse the loss of vision. A critical barrier for developing therapy for this group of diseases is their heterogeneous nature involving many pathways and genes and therefore developing targeted therapy (e.g. gene therapy) is practically impossible for each gene or mutation. We searched for a common mediator or a ‘second messenger’ that involves in the process of photoreceptor cell death, which can be targeted to prevent or delay the diseases progression. We and others identified sphingolipid, ceramide, a potent mediator of cell death, generated either from a dyeing cell or produced abnormally in diseased tissue, increases in the retina during cell death. Irrespective of the causal factor (genes or mutations), ceramide could therefore be targeted for preventing and delaying photoreceptor cell death. I will discuss our progress in this journey of targeting ceramide for photoreceptor cell death using animal models of human retinal degenerations.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.
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