诱导多能干细胞作为治疗儿科患者个性化的模型：疾病模型和药物不良反应预防

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

Volume: 25 Issue: 24

关键词: 诱导多能干细胞，疾病模型，药物不良反应，儿科患者，治疗个性化，创新药理学模型，肝毒性，胰腺炎。

摘要: 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.

Cite this article as:

Induced Pluripotent Stem Cells as a Model for Therapy Personalization of Pediatric Patients: Disease Modeling and Drug Adverse Effects Prevention, Current Medicinal Chemistry 2018; 25 (24) . https://dx.doi.org/10.2174/0929867324666170804150131