Understanding the biological and molecular processes underlying human pathologies
is fundamental in order to develop innovative approaches to treat or prevent
them. Among the technologies that could provide innovative disease models, induced
pluripotent stem cells (iPSCs) is one of the most promising. Indeed, one application of
this technology is patient-specific disease modeling. iPSCs obtained by reprogramming
patients’ cells collected from accessible tissues, have the unique capability to differentiate,
under an adequate stimulus, into any human cell type. In particular, iPSCs technology
can be applied to study drug adverse effects, that is a key part of the drug discovery process.
Indeed, drug induced adverse effects are among the most common causes that lead to
abandon the development of new candidate therapeutic molecules, increasing the cost of
drug discovery. An innovative strategy that could be used in drug design to solve drug attrition
rate, and to establish innovative pharmacological models, could be the application
of iPSCs technology in the early stage of the drug discovery process to model druginduced
adverse events. In this review, recently developed disease models based on iPSCs
will be discussed, with a particular focus on available models of drugs’ adverse effect, in
particular hepatic/pancreatic toxicity.
Keywords: Induced pluripotent stem cells, disease modeling, drug adverse effects, pediatric patients, therapy personalization,
innovative pharmacological models, hepatotoxicity, pancreatitis.
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