Integration of human stem cell-derived in vitro systems and mouse preclinical models identifies complex pathophysiologic mechanisms in retinal dystrophy [human_retinal_organoid]

We have optimized CRISPR/Cas genome editing in human Embryonic Stem Cells (hESCs) and developed a method to generate hESC-derived eye organoids to investigate the cell type composition of these eye organoids using single cell RNA sequencing. We also investigate the consequences of the loss of a new AMD-associated gene, DRAM2, on the cell composition of the eye organoids. 2 samples from eye organoids. 1 organoid sample derived from DRAM2 WT stem cells with three replicates (as controls); 1 organoid sample derived from DRAM2 KO stem cells with four replicates. All samples sequenced for single cell RNAseq