Despite some technical limits, CRISPR/Cas9-based genome editing of hiPSC is a powerful approach to elucidate gene function in congenital pituitary deficiency patients, as was shown for the role of hypothalamic OTX2 regulation of pituitary progenitor cells in congenital pituitary hypoplasia (Matsumoto et al., 2019). This evidence concerns the gene OTX2 and pituitary deficiency.