Human Genetics

, Volume 135, Issue 9, pp 1071–1082 | Cite as

Genome editing and the next generation of antiviral therapy

  • Daniel Stone
  • Nixon Niyonzima
  • Keith R. Jerome
Part of the following topical collections:
  1. Genome Editing


Engineered endonucleases such as homing endonucleases (HEs), zinc finger nucleases (ZFNs), Tal-effector nucleases (TALENS) and the RNA-guided engineered nucleases (RGENs or CRISPR/Cas9) can target specific DNA sequences for cleavage, and are proving to be valuable tools for gene editing. Recently engineered endonucleases have shown great promise as therapeutics for the treatment of genetic disease and infectious pathogens. In this review, we discuss recent efforts to use the HE, ZFN, TALEN and CRISPR/Cas9 gene-editing platforms as antiviral therapeutics. We also discuss the obstacles facing gene-editing antiviral therapeutics as they are tested in animal models of disease and transition towards human application.


Gene Editing Viral Episome Antiviral Therapeutics Engineer Endonuclease 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.



We thank Chelsea Spragg for critical reading of this manuscript. This work was funded by a philanthropic Grant from the Caladan Foundation, by NIH supported Martin Delaney Collaboratory Grant U19 AI 096111, NIH Grants R21 AI117519 and R21 AI107252, and in part by a developmental Grant from the University of Washington Center for AIDS Research (CFAR), an NIH funded program under Award Number P30 AI 027757 which is supported by the following NIH Institutes and Centers (NIAID, NCI, NIMH, NIDA, NICHD, NHLBI, NIA, NIGMS, NIDDK).

Compliance with ethical standards

Conflict of interest

The authors declare no conflict of interest.


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Copyright information

© Springer-Verlag Berlin Heidelberg 2016

Authors and Affiliations

  • Daniel Stone
    • 1
  • Nixon Niyonzima
    • 1
    • 3
  • Keith R. Jerome
    • 1
    • 2
  1. 1.Vaccine and Infectious Disease DivisionFred Hutchinson Cancer Research CenterSeattleUSA
  2. 2.Department of Laboratory MedicineUniversity of WashingtonSeattleUSA
  3. 3.Graduate Program in Molecular and Cellular BiologyUniversity of WashingtonSeattleUSA

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