Abstract
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.
| Original language | English |
|---|---|
| Journal | Current Medicinal Chemistry |
| DOIs | |
| Publication status | E-pub ahead of print - Aug 4 2017 |
Fingerprint
Cite this
Induced pluripotent stem cells as a model for therapy personalization of pediatric patients : disease modeling and drug adverse effects prevention. / Genova, Elena; Pelin, Marco; Sasaki, Katsunori; Fengming, Yue; Lanzi, Gaetana; Masneri, Stefania; Ventura, Alessandro; Stocco, Gabriele; Decorti, Giuliana.
In: Current Medicinal Chemistry, 04.08.2017.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Induced pluripotent stem cells as a model for therapy personalization of pediatric patients
T2 - disease modeling and drug adverse effects prevention
AU - Genova, Elena
AU - Pelin, Marco
AU - Sasaki, Katsunori
AU - Fengming, Yue
AU - Lanzi, Gaetana
AU - Masneri, Stefania
AU - Ventura, Alessandro
AU - Stocco, Gabriele
AU - Decorti, Giuliana
N1 - Copyright© Bentham Science Publishers; For any queries, please email at epub@benthamscience.org.
PY - 2017/8/4
Y1 - 2017/8/4
N2 - 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.
AB - 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.
U2 - 10.2174/0929867324666170804150131
DO - 10.2174/0929867324666170804150131
M3 - Article
C2 - 28782468
JO - Current Medicinal Chemistry
JF - Current Medicinal Chemistry
SN - 0929-8673
ER -