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CRISPR-Cas9: A Precise Approach to Genome Engineering

Therapeutic Innovation & Regulatory Science volume 52, pages 701–707 (2018)Cite this article

Abstract

In the last few decades, genomic manipulation has made significant progress as a result of the development of recombinant DNA technologies; however, more often than not, these techniques have been costly and labor intensive. In contrast, recently developed next-generation sequencing (NGS) technologies have provided a cheaper, faster, and easier process to study genomics. In particular, an NGS technique emerged from bacterial CRISPR-associated protein-9 nuclease (Cas9) as a revolutionary method to modify, regulate, or mark specific genomic sequences on virtually any organism. A later adaptation of this bacterial defense mechanism that successfully and permanently edits dysfunctional genes and corrects missing proteins has resulted in a new era for disease genetic engineering. Clinical trials using this technique are already being performed, and the applicability of CRISPR-Cas9 techniques is actively being investigated using in vivo studies. However, the concept of genome correction poses great concerns from a regulatory perspective, especially in terms of security, so principles for the regulation of these methodologies are being established. We delved into CRISPR-Cas9 from its natural and ortholog origins to its engineered variants and behaviors to present its notable and diverse applications in the fields of biotechnology and human therapeutics.

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Affiliations

  1. School of Biological Sciences and Engineering, Yachay Tech University, Urcuquí, Ecuador

    Jorge E. Simón, Ángel S. Rodríguez & Nelson Santiago Vispo PhD

  2. San Miguel de Urcuquí, Hacienda San José s/n, Urcuquí, Ecuador

    Nelson Santiago Vispo PhD

Authors
  1. Jorge E. Simón
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  2. Ángel S. Rodríguez
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  3. Nelson Santiago Vispo PhD
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Correspondence to Nelson Santiago Vispo PhD.

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Simón, J.E., Rodríguez, Á.S. & Santiago Vispo, N. CRISPR-Cas9: A Precise Approach to Genome Engineering. Ther Innov Regul Sci 52, 701–707 (2018). https://doi.org/10.1177/2168479018762798

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Keywords

  • CRISPR-Cas9
  • NGS
  • bacterial adaptive defense system
  • genetic engineering
  • clinical trials