Mesenchymal drift in ciliopathy iPSC-derived RPE reveals a convergent pathogenic cell state.
This study develops induced pluripotent stem cell-derived RPE models (iRPE) and identifies two drugs, pioglitazone, a mitochondrial metabolic modulator, and galunisertib, a TGFBR1 inhibitor, as potential therapeutic candidates for multiple ciliopathy subtypes.