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Article

Nicotinamide Ameliorates Disease Phenotypes in a Human iPSC Model of Age-Related Macular Degeneration Graphical Abstract

Authors Janmeet S. Saini, Barbara Corneo, Justine D. Miller, ..., Timothy A. Blenkinsop, Jeffrey H. Stern, Sally Temple

Correspondence sallytemple@neuralsci.org

In Brief Saini et al. show that hiPSC-derived RPE, including ARMS2/HTRA1 homozygotes, from AMD patients exhibit higher complement and inflammatory factors compared to healthy controls. Nicotinamide treatment reduces these and other AMD-related molecules with no observed cytotoxicity. Pursuing nicotinamide’s mechanism of action should reveal new therapeutic approaches for AMD.

Highlights d

Complement and inflammatory factors are upregulated in AMD hiPSC-derived RPE

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Nicotinamide inhibits AMD biomarkers, including drusen components and VEGFA

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Nicotinamide suppresses production of complement and inflammatory factors

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Nicotinamide increases RPE cell survival while targeting aging-associated pathways

Saini et al., 2017, Cell Stem Cell 20, 635–647 May 4, 2017 ª 2017 Elsevier Inc. http://dx.doi.org/10.1016/j.stem.2016.12.015

Data Resources GSE90889


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