Induced Pluripotent Stem Cells as a Model for Therapy Personalization of Pediatric Patients: Disease Modeling and Drug Adverse Effects Prevention

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Author(s): Elena Genova, Marco Pelin, Katsunori Sasaki*, Yue Fengming, Gaetana Lanzi, Stefania Masneri, Alessandro Ventura, Gabriele Stocco, Giuliana Decorti.

Journal Name: Current Medicinal Chemistry


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 iPSCs technology is personalized disease modeling. These cells, 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 (human) drug toxicity, 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 drug-induced 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 pluripotents stem cells, disease modeling, drug adverse effects, pediatric patients

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(E-pub Ahead of Print)
DOI: 10.2174/0929867324666170804150131
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