Human induced pluripotent stem cells illuminate pathways and novel treatment targets for age-related macular degeneration

A systems biology approach towards understanding and treating non-neovascular age-related macular degeneration

“…Saini et al 94 also found a graded response to nicotinamide (NAM), with a marked inhibition of drusen-associated proteins, clusterin and vascular endothelial growth factor A, in iPS-RPE cells with the ARMS2/HTRA1 AMD risk variant. These experiments underscore the importance of targeting more than one disease pathway or mechanism at a time, while taking into consideration genotype risk 95 . Alternatively, the emergence of CRISPR/Cas9 gene editing has enabled researchers to further manipulate stem cell systems for mechanistic study.…”