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Gene expression of renal cells in kidney organoids, such as podocytes and tubular epithelial cells, was increased after ERT in GLA-KO human iPSC kidney organoids (Fig
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To investigate this point for retinopathies, we used two different animal models: (i) young and aged mice treated with NACET to evaluate the effect of the drug on retinal aging as a model for events triggering the AMD phenotype, and (ii) Akita mice as a characterized and robust model for DR
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