Human disease modeling with induced pluripotent stem cells

Alan Trounson, Kelly A. Shepard, Natalie D DeWitt

Research output: Contribution to journalReview ArticleResearchpeer-review

37 Citations (Scopus)


In the past few years, cellular programming, whereby virtually all human cell types, including those deep within the brain or internal organs, can potentially be produced and propagated indefinitely in culture, has opened the door to a new type of disease modeling. Importantly, many diseases or disease predispositions have genetic components that vary from person to person. Now cells from individuals can be readily reprogrammed to form pluripotent cells, and then directed to differentiate into the lineage and the cell type in which the disease manifests. Those cells will contain the genetic contribution of the donor, providing an excellent model to delve into human disease at the level of individuals and their genomic variants. To date, over fifty such disease models have been reported, and while the field is young and hurdles remain, these tools promise to inform scientists about the cause and cellular-molecular mechanisms involved in pathology, unravel the role of environmental versus hereditary factors driving disease, and provide an unprecedented tool for screening therapeutic agents that might slow or halt disease progression.

Original languageEnglish
Pages (from-to)509-516
Number of pages8
JournalCurrent Opinion in Genetics and Development
Issue number5
Publication statusPublished - Oct 2012
Externally publishedYes

Cite this