We subsequently tested the potential of two small molecule compounds, 4-phenyl butyric acid (4-PBA) and 5-aminoimidazole-4-carboxamide ribonucleotide (AICAR), previously identified for their ability to enhance ABCA4 missense variants trafficking to the cell surface in vitro,26 to improve ABCA4 protein traffic in retinal organoids and evaluate their feasibility as a missense-variant–independent therapeutic approach for the treatment of ABCA4-associated retinopathy. The gene discussed is ABCA4; the disease is retinal disorder.