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

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World Journal of Stem Cells 2019年第12期11卷 1020-1044页

作者： Elena Genova federica cavion Marianna Lucafò Luigina De Leo Marco Pelin Gabriele Stocco Giuliana Decorti PhD School in Reproduction and Development Sciences University of TriesteTrieste 34127Italy Department of Life Sciences University of TriesteTrieste 34127Italy Institute for Maternal and Child Health IRCCS Burlo GarofoloTrieste 34137Italy Department of Medical Surgical and Health SciencesUniversity of TriesteTrieste 34127Italy

Adverse drug reactions(ADRs)are major clinical problems,particularly in special populations such as pediatric ***,ADRs may be caused by a plethora of different drugs leading,in some cases,to hospitalization,disability or even *** 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 *** improve the comprehension,and thus the prevention,of ADRs,the set-up of sensitive and personalized assays is urgently *** progress is represented by the possibility of setting up groundbreaking patient-specific *** 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 *** 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 *** especially focused on the intestinal,hepatic,pancreatic,renal,cardiac,and neuronal levels,also discussing progress in organoids *** 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 *** on the existing knowledge,these models are powerful and promising tools in multiple clinical applications including toxicity screening,disease modeling,personalized and regenerative medicine.

关键词： Induced pluripotent stem cells Organoids Adverse drug reactions Intestinal toxicity Hepatic toxicity Pancreatic toxicity Nephrotoxicity Cardiotoxicity Neurotoxicity

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