Modelling Human Disease with Pluripotent Stem Cells

Title:Modelling Human Disease with Pluripotent Stem Cells

Volume: 13 Issue: 2

Author(s): Richard Siller, Sebastian Greenhough, In-Hyun Park and Gareth J. Sullivan

Affiliation:

Keywords: Induced pluripotent stem cells, human embryonic stem cells, neurodevelopmental disorders, endodermal disorders, mesodermal disorders, reprogramming, disease modelling

Abstract: Recent progress in the field of cellular reprogramming has opened up the doors to a new era of disease modelling, as pluripotent stem cells representing a myriad of genetic diseases can now be produced from patient tissue. These cells can be expanded and differentiated to produce a potentially limitless supply of the affected cell type, which can then be used as a tool to improve understanding of disease mechanisms and test therapeutic interventions. This process requires high levels of scrutiny and validation at every stage, but international standards for the characterisation of pluripotent cells and their progeny have yet to be established. Here we discuss the current state of the art with regard to modelling diseases affecting the ectodermal, mesodermal and endodermal lineages, focussing on studies which have demonstrated a disease phenotype in the tissue of interest. We also discuss the utility of pluripotent cell technology for the modelling of cancer and infectious disease. Finally, we spell out the technical and scientific challenges which must be addressed if the field is to deliver on its potential and produce improved patient outcomes in the clinic.

Cite this article as:

Siller Richard, Greenhough Sebastian, Park In-Hyun and J. Sullivan Gareth, Modelling Human Disease with Pluripotent Stem Cells, Current Gene Therapy 2013; 13 (2) . https://dx.doi.org/10.2174/1566523211313020004