Induced pluripotent stem cells for therapy personalization in pediatric patients: Focus on drug-induced adverse events

Elena Genova, Federica Cavion, Marianna Lucafò, Luigina De Leo, Marco Pelin, Gabriele Stocco, Giuliana Decorti

Research output: Contribution to journalReview articlepeer-review


Adverse drug reactions (ADRs) are major clinical problems, particularly in special populations such as pediatric patients. Indeed, ADRs may be caused by a plethora of different drugs leading, in some cases, to hospitalization, disability or even death. In addition, pediatric patients may respond differently to drugs with respect to adults and may be prone to developing different kinds of ADRs, leading, in some cases, to more severe consequences. To improve the comprehension, and thus the prevention, of ADRs, the set-up of sensitive and personalized assays is urgently needed. Important progress is represented by the possibility of setting up groundbreaking patient-specific assays. This goal has been powerfully achieved using induced pluripotent stem cells (iPSCs). Due to their genetic and physiological species-specific differences and their ability to be differentiated ideally into all tissues of the human body, this model may be accurate in predicting drug toxicity, especially when this toxicity is related to individual genetic differences. This review is an up-to-date summary of the employment of iPSCs as a model to study ADRs, with particular attention to drugs used in the pediatric field. We especially focused on the intestinal, hepatic, pancreatic, renal, cardiac, and neuronal levels, also discussing progress in organoids creation. The latter are three-dimensional in vitro culture systems derived from pluripotent or adult stem cells simulating the architecture and functionality of native organs such as the intestine, liver, pancreas, kidney, heart, and brain. Based on the existing knowledge, these models are powerful and promising tools in multiple clinical applications including toxicity screening, disease modeling, personalized and regenerative medicine.

Original languageEnglish
Pages (from-to)1020-1044
Number of pages25
JournalWorld Journal of Stem Cells
Issue number12
Publication statusPublished - Dec 1 2019


  • Adverse drug reactions
  • Cardiotoxicity
  • Hepatic toxicity
  • Induced pluripotent stem cells
  • Intestinal toxicity
  • Nephrotoxicity
  • Neurotoxicity
  • Organoids
  • Pancreatic toxicity

ASJC Scopus subject areas

  • Histology
  • Molecular Biology
  • Genetics
  • Genetics(clinical)
  • Cell Biology


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