International Journal of

ADVANCED AND APPLIED SCIENCES

EISSN: 2313-3724, Print ISSN: 2313-626X

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 Volume 7, Issue 10 (October 2020), Pages: 20-29

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 Review Paper

 Title: CRISPR/Cas9: The new era of gene therapy

 Author(s): Amal Alotaibi *

 Affiliation(s):

 Department of Basic Health Science, College of Medicine, Princess Nourah Bint Abdulrahman University, Riyadh, Saudi Arabia

  Full Text - PDF          XML

 * Corresponding Author. 

  Corresponding author's ORCID profile: https://orcid.org/0000-0001-9819-447X

 Digital Object Identifier: 

 https://doi.org/10.21833/ijaas.2020.10.003

 Abstract:

The Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein (CRISPR/Cas) system is present in many Bacteria and in almost all Archaea and functions in those organisms as a defense mechanism against invading viruses and plasmids. Since the function and the working mechanism of the bacterial CRISPR/Cas system were elucidated in 2007, and researchers realized its potential as a gene-editing tool in 2012, it quickly became a widely used tool to generate mutations in cells, cell lines, and various model organisms. Applications such as improvement of disease resistance of economic plants, enhancement of muscle growth, or litter size in livestock are already becoming common practice. More recently, its application to repair genetic mutations has been explored in human cells and cell lines, and currently, the first clinical trials are underway in which the CRISPR/Cas9 system is applied to cure patients from various diseases. When looking at the timeline, it is clear that the CRISPR/Cas9 system has revolutionized genome engineering in less than a decade and may well be the most versatile genome engineering tool available. Here we review the origin and function of the CRISPR/Cas9 system, its working mechanism, and its use in various model organisms. In addition, pitfalls of the method are discussed, as well as the currently running and planned clinical trials. The objectives of this review are: first, to inform readers of the working mechanism of this new technique and how it is currently used to facilitate research in model organisms and to improve the profitability of livestock and economic plants. And second, to provide insight into the application of this technique in the treatment of disease in humans. 

 © 2020 The Authors. Published by IASE.

 This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

 Keywords: CRISPR/Cas9, Gene therapy, Gene editing

 Article History: Received 9 February 2020, Received in revised form 10 June 2020, Accepted 11 June 2020

 Acknowledgment:

This research was funded by the Deanship of Scientific Research at Princess Nourah, bint Abdulrahman University through the Fast-track Research Funding Program.

 Compliance with ethical standards

 Conflict of interest: The authors declare that they have no conflict of interest.

 Citation:

 Alotaibi A (2020). CRISPR/Cas9: The new era of gene therapy. International Journal of Advanced and Applied Sciences, 7(10): 20-29

 Permanent Link to this page

 Figures

 Fig. 1 Fig. 2

 Tables

 Table 1

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