Among monogenic retinal disorders, BEST1-associated retinopathies provide a particularly informative paradigm for linking molecular mechanisms to emerging regenerative strategies.The human BEST1 encodes bestrophin-1 (BEST1), a calcium-activated chloride channel predominantly expressed in the RPE, where it plays essential roles in ionic homeostasis, transepithelial transport, and regulation of the visual cycle. This evidence concerns the gene BEST1 and retinal disorder.