Notably, loss of Best1 function in patient-derived iPSC-RPE cells can be fully rescued by AAV2-mediated WT BEST1 gene augmentation [117], while gain-of-function BEST1 mutations are also rescuable by a combination of gene augmentation with CRISPR/Cas9-mediated knockdown of endogenous Best1 expression [118], underlying a bright future of stem cell- and/or gene-based therapies for the treatment of bestrophinopathies. This evidence concerns the gene BEST1 and autosomal recessive bestrophinopathy.