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, 48 (5), 256-65

iPSC technology--Powerful Hand for Disease Modeling and Therapeutic Screen


iPSC technology--Powerful Hand for Disease Modeling and Therapeutic Screen

Changsung Kim. BMB Rep.


Cardiovascular and neurodegenerative diseases are major health threats in many developed countries. Recently, target tissues derived from human embryonic stem (hES) cells and induced pluripotent stem cells (iPSCs), such as cardiomyocytes (CMs) or neurons, have been actively mobilized for drug screening. Knowledge of drug toxicity and efficacy obtained using stem cell-derived tissues could parallel that obtained from human trials. Furthermore, iPSC disease models could be advantageous in the development of personalized medicine in various parts of disease sectors. To obtain the maximum benefit from iPSCs in disease modeling, researchers are now focusing on aging, maturation, and metabolism to recapitulate the pathological features seen in patients. Compared to pediatric disease modeling, adult-onset disease modeling with iPSCs requires proper maturation for full manifestation of pathological features. Herein, the success of iPSC technology, focusing on patient-specific drug treatment, maturation-based disease modeling, and alternative approaches to compensate for the current limitations of patient iPSC modeling, will be further discussed.


Fig. 1.
Fig. 1.. Schematic view of novel hES/iPSC-derived CM-based human cardiac toxicity test. Ikr (rapid component of inward rectifying K+ current, hERG) is the major category to correlate possible human cardio toxicity. Decrease of hERG by drugs indicating the increase of QT interval by drugs, which induce severe cardiotoxic event.
Fig. 2.
Fig. 2.. Human patients and patient gene edited iPSC/hES-mediated novel therapy development. Disease containing stem cells were differentiated into target tissue such as neurons and CM. Adult-stage-maturated target tissue will be used to screen novel therapeutic candidates or market-available drugs to find right dosage for individual patients. Novel drugs’ cardiotoxicity test will be performed while optimizing the efficacy of target hit. Optimized therapy will be pursued to live human patients.

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