iPS cell-derived models offer new opportunities for drug screening

New models for Down syndrome and amyotrophic lateral sclerosis harness patient-derived induced pluripotent stem (iPS) cells to overcome genetic and cell heterogeneities inherent to the disorders not adequately recapitulated by cell culture and animal models.

In a Science Advances study, a group from Massachusetts Institute of Technology and Harvard University developed a 3D model of ALS by culturing iPS cell-derived muscle bundles and patient-derived iPS motor neurons in linked chambers on an organ-on-a-chip. The iPS cell-derived muscles and neurons formed neuromuscular junctions with weak muscle contractions and neuronal cell death similar to pathologies observed in ALS patients...