Molecular Biotechnology

, Volume 60, Issue 4, pp 329–338 | Cite as

Genome Editing in Stem Cells for Disease Therapeutics

  • Minjung SongEmail author
  • Suresh RamakrishnaEmail author


Programmable nucleases including zinc finger nucleases, transcription activator-like effector nucleases, and clustered regularly interspaced short palindrome repeats (CRISPR)/CRISPR-associated protein have tremendous potential biological and therapeutic applications as novel genome editing tools. These nucleases enable precise modification of the gene of interest by disruption, insertion, or correction. The application of genome editing technology to pluripotent stem cells or hematopoietic stem cells has the potential to remarkably advance the contribution of this technology to life sciences. Specifically, disease models can be generated and effective therapeutics can be developed with great efficiency and speed. Here we review the characteristics and mechanisms of each programmable nuclease. In addition, we review the applications of these nucleases to stem cells for disease therapies and summarize key studies of interest.


Zinc finger nucleases Transcription activator-like effector nucleases Clustered regularly interspaced short palindrome repeat associated system Induced pluripotent stem cells Hematopoietic stem cells 



This research was supported by the Basic Science Research Program through the National Research Foundation of Korea (NRF), which is funded by the Ministry of Education (NRF-2017R1D1A1A02018480 and 2017M3A9C6061361). Medical Research Center (2017R1A5A2015395), funded by the National Research Foundation of Korea (NRF) of the Ministry of Science, ICT and Future Planning, Republic of Korea and Bio and Medical Technology Development Program of the National Research Foundation (NRF) and funded by the Korean government (MSIP and MOHW) (2017M3A9E4048172). The author would like to thank Ms. Hee-Jung Seo and Jisu Song from Silla University for their figure illustrations. The authors declare no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.


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© Springer Science+Business Media, LLC, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Department of Food Biotechnology, College of Medical and Life ScienceSilla UniversityBusanSouth Korea
  2. 2.Graduate School of Biomedical Science and EngineeringHanyang UniversitySeoulSouth Korea
  3. 3.College of MedicineHanyang UniversitySeoulSouth Korea

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