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Cell-autonomous lipid-handling defects in Stargardt iPSC-derived retinal pigment epithelium cells

27 Oct 2022

Abstract excerpt

Stargardt retinopathy is an inherited form of macular degeneration caused by mutations in gene ABCA4 and characterized by the accumulation of lipid-rich deposits in the retinal pigment epithelium (RPE), RPE atrophy, and photoreceptor cell death. Inadequate mechanistic insights into pathophysiological changes occurring in Stargardt RPE have hindered disease treatments. Here, we show that ABCA4 knockout and induced...

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PMID: 36306781
DOI: 10.1016/j.stemcr.2022.10.001

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