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Table 1 Latest hiPSCs drug screening

From: Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery

Research field hiPSCs-derived cells Drugs tested Outcome Ref
Colon cancer Cardiomyocytes and endothelial cells, cocultured with tumor spheroids 2 anticancer drugs were tested in order to simultaneously assess cardiac toxicity and antitumor effects Demonstration of the feasibility to simultaneously assess cardiac toxicity and antitumor drugs effects [16]
Lesch-Nyhan disease (OMIM #300322) Cortical neurons Test of 3838 compounds Identification of 6 pharmacological compounds correcting phosphoribosyltransferase (HGPRT) deficiency–associated neuronal phenotypes [17]
Progressive fibrosis Mesenchymal-like cells Libraries of ~ 17000 small molecules Identification of anti-fibrotic small molecule [18]
Neuroprotective activity of pharmacological compounds Embryoid bodies, neuronal precursors and neurons Screening of peptides of the melanocortin family and endocannabinoids for cytotoxicity, embryotoxicity, and neuroprotective potential Both melanocortin peptides and endocannabinoids exerted neuroprotective effects [19]
Pro-regenerative drug development Cardiomyocytes Test of 150 small molecules with pro-regenerative potential Identification of 2 pro-proliferative compounds acting via the mevalonate pathway. [20]
Alzheimer’s disease (OMIM #104300) Neurons Test of R33 molecule R33 is able to reduce Aβ and pTAU [21]
Schizophrenia (SZ) Neural progenitor cells Screening of 135 drugs with predicted or known interactions in SZ-biology Identification of 52 drugs ameliorating the SZ-related transcriptomic signature hiPSCs-derived neural progenitor cells [22]
Amyotrophic lateral sclerosys (ALS) (OMIM #105400) Motor neurons 1416 compounds involved in motor neurons survival Identification of Src/c-Abl inhibitors increasing the survival rate of ALS motor neurons [23]
Homozygous familial hypercholesterolemia (OMIM #144010) Hepatocytes Screening of a library of 2320 existing drugs Identification of 5 cardiac glycosides able to reduce the hepatocytes production of apoB [24]
Long-QT syndrome Cardiomyocytes Test of LUF7346, known hERG allosteric modulators LUF7346 is able to rescue some phenotypic features of Long-QT-syndrome [25]