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Therapeutic applications of CRISPR/Cas9 system in gene therapy

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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.

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Authors and Affiliations

  1. Department of Medical Genetics, Iran University of Medical Sciences (IUMS), Crossroads of Shahid Hemmat & Shahid Chamran Highways, P.O. Box: 15875-6171, 1449614535, Tehran, Iran

    Hasan Mollanoori & Shahram Teimourian

  2. Department of Infectious Diseases, School of Medicine, Pediatric Infectious Diseases Research Center, Tehran University of Medical Sciences, Tehran, Iran

    Shahram Teimourian

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  1. Hasan Mollanoori
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  2. Shahram Teimourian
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Correspondence to Shahram Teimourian.

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Mollanoori, H., Teimourian, S. Therapeutic applications of CRISPR/Cas9 system in gene therapy. Biotechnol Lett 40, 907–914 (2018). https://doi.org/10.1007/s10529-018-2555-y

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  • DOI: https://doi.org/10.1007/s10529-018-2555-y

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