Pluripotent stem cells in disease modelling and drug discovery

Yishai Avior, Ido Sagi, Nissim Benvenisty*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

434 Scopus citations


Experimental modelling of human disorders enables the definition of the cellular and molecular mechanisms underlying diseases and the development of therapies for treating them. The availability of human pluripotent stem cells (PSCs), which are capable of self-renewal and have the potential to differentiate into virtually any cell type, can now help to overcome the limitations of animal models for certain disorders. The ability to model human diseases using cultured PSCs has revolutionized the ways in which we study monogenic, complex and epigenetic disorders, as well as early- and late-onset diseases. Several strategies are used to generate such disease models using either embryonic stem cells (ES cells) or patient-specific induced PSCs (iPSCs), creating new possibilities for the establishment of models and their use in drug screening.

Original languageAmerican English
Pages (from-to)170-182
Number of pages13
JournalNature Reviews Molecular Cell Biology
Issue number3
StatePublished - 1 Mar 2016

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