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Biotechnology Letters

, Volume 40, Issue 6, pp 907–914 | Cite as

Therapeutic applications of CRISPR/Cas9 system in gene therapy

  • Hasan Mollanoori
  • Shahram TeimourianEmail author
Review

Abstract

Gene therapy is based on the principle of the genetic manipulation of DNA or RNA for treating and preventing human diseases. The clustered regularly interspaced short palindromic repeats/CRISPR associated nuclease9 (CRISPR/Cas9) system, derived from the acquired immune system in bacteria and archaea, has provided a new tool for accurate manipulation of genomic sequence to attain a therapeutic result. The advantage of CRISPR which made it an easy and flexible tool for diverse genome editing purposes is that a single protein (Cas9) complex with 2 short RNA sequences, function as a site-specific endonuclease. Recently, application of CRISPR/Cas9 system has become popular for therapeutic aims such as gene therapy. In this article, we review the fundamental mechanisms of CRISPR-Cas9 function and summarize preclinical CRISPR-mediated gene therapy reports on a wide variety of disorders.

Keywords

CRISPR/cas9 Gene therapy 

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Copyright information

© Springer Science+Business Media B.V., part of Springer Nature 2018

Authors and Affiliations

  1. 1.Department of Medical GeneticsIran University of Medical Sciences (IUMS)TehranIran
  2. 2.Department of Infectious Diseases, School of Medicine, Pediatric Infectious Diseases Research CenterTehran University of Medical SciencesTehranIran

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