NR2E1 and Sandhoff disease: Moreover, the top nine upregulated genes in isogenic HEXB-corrected organoids relative to Sandhoff disease organoids encoded transcription factors critical in neuron morphogenesis and central nervous system development: TRB1 (34), FOXG1 (35), NR2E1 (36), FEZF2 (37), NEUROD2 (38), OTX1 (39), EMX1 (40), EOMES (41), and LHX2 (42) (Fig. 9C).