Cellular Engineering and Disease Modeling with Gene-Editing Nucleases

Research output: Chapter in Book/Report/Conference proceedingChapter

1 Scopus citations


Two rapidly evolving technologies are set to intersect at the crossroads of the future of medicine: the knowledge of how to induce and maintain cellular pluripotency, and the ability to precisely manipulate the genome with engineered nucleases. Together, these two advances have significant potential in the development of the next generation of cell and gene therapies. This review will discuss human and animal models of stem cells and the application of engineered nucleases for precision gene targeting and control. For animal studies and models, nucleases have allowed for greater flexibility and expandability. Previously untargetable regions of the murine genome are now accessible via engineered nucleases. Prior to the availability of gene editing proteins, the entire rat genome was largely refractory to gene targeting and manipulation. The ability to engineer larger animals may reduce the transplant organ gap and increase the yields of food for an expanding population. Lastly, the ability to modify stem cells of hematopoietic, embryonic, or somatic origin will allow for more relevant disease modeling, and more targeted and effective therapies. Collectively, the efficiency of gene editing nucleases and the ability to apply them across cells of multiple species allows for new research opportunities, more flexibility, and greater accuracy in choosing the model best suited for genome manipulation.

Original languageEnglish (US)
Title of host publicationAdvances in Experimental Medicine and Biology
Number of pages36
StatePublished - 2016

Publication series

NameAdvances in Experimental Medicine and Biology
ISSN (Print)0065-2598
ISSN (Electronic)2214-8019

Bibliographical note

Publisher Copyright:
© American Society of Gene and Cell Therapy 2016.


  • Clustered regularly-interspaced short palindromic repeats (CRISPR)/ Cas9
  • Embryonic stem cell (ESC)
  • Hematopoietic stem cell (HSC)
  • Homologous recombination (HR)
  • Inducible pluripotent stem cell (iPSC)
  • Insertions/deletions (indels)
  • Meganuclease (MN)
  • Non-homologous end joining (NHEJ)
  • Oligonucleotide donor (ODN)
  • Reprogramming
  • Somatic cell nuclear transfer (SCNT)
  • Transcription activator-like effector nuclease (TALEN)
  • Zinc finger nuclease (ZFN)


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