iPSC-derived human venous endothelial cells for drug discovery
There is a lack of robust disease models for vascular malformations (VMs) for drug discovery.
The researchers generate lineage-specific human induced VECs (iVECs) through manipulation of cell-cycle dynamics via the retinoic signaling pathway.
The authors also introduced a heterozygous TIE2-mutation in human iPSCs and transplanting these mutated iVECs recapitulate venous malformations in vivo.
Deep learning combined with DRUG-seq is then used to pinpoint small molecules that can reverse the symptoms of vascular malformations.
https://www.cell.com/cell-stem-cell/fulltext/S1934-5909(24)00377-1