Current Stem Cell Reports

, Volume 4, Issue 3, pp 253–263 | Cite as

Gene Editing of Stem Cells to Model and Treat Disease

  • Jennifer A. HollywoodEmail author
  • David J. Sanz
  • Alan J. Davidson
  • Patrick T. Harrison
Genome Editing (SN Waddington and HC O'Neill, Section Editors)
Part of the following topical collections:
  1. Topical Collection on Genome Editing


Purpose of Review

The ability to reprogramme any adult cell back into a pluripotent stem cell state coupled with the capability to precisely edit its genomic sequence has given scientists almost unlimited potential to model any genetic disease and holds great promise for regenerative medicine.

Recent Findings

The discovery of CRISPR/Cas9 coupled with continued development and modification of this system has made it possible to manipulate gene expression at both a genetic and epigenetic level exposing the true power of stem cells. The applications of this technology to model any genetic disease, screen new drugs, generate organs for donation and develop cell therapies appear limitless.


In this review, we will provide a SWOT analysis of CRISPR/Cas9 and discuss the most recent developments in induced pluripotent stem cell gene editing and the impact being made in the clinic.


Stem cells Induced pluripotent stem cells CRISPR/Cas9 Gene editing Disease modelling Regenerative medicine 


Compliance with Ethical Standards

Conflict of Interest

Jennifer A. Hollywood, David J. Sanz, Alan J. Davidson, and Patrick T. Harrison declare that they have no conflict of interest.

Human and Animal Rights and Informed Consent

Ethical Approval

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.


Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Copyright information

© Springer Nature Switzerland AG 2018

Authors and Affiliations

  • Jennifer A. Hollywood
    • 1
    Email author
  • David J. Sanz
    • 1
    • 2
  • Alan J. Davidson
    • 1
  • Patrick T. Harrison
    • 1
    • 2
  1. 1.Department of Molecular Medicine & PathologyUniversity of AucklandAucklandNew Zealand
  2. 2.Department of PhysiologyUniversity College CorkCorkIreland

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