CRISPR-Cas9: A Precise Approach to Genome Engineering

Ther Innov Regul Sci. 2018 Nov;52(6):701-707. doi: 10.1177/2168479018762798. Epub 2018 Apr 2.


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.

Keywords: CRISPR-Cas9; NGS; bacterial adaptive defense system; clinical trials; genetic engineering.

Publication types

  • Review

MeSH terms

  • Bacterial Proteins / metabolism
  • CRISPR-Associated Protein 9 / metabolism*
  • CRISPR-Cas Systems
  • Clinical Trials as Topic / legislation & jurisprudence
  • Genetic Engineering / legislation & jurisprudence*
  • Genetic Engineering / methods
  • Genome, Human
  • High-Throughput Nucleotide Sequencing
  • Humans
  • Mutation
  • Sequence Analysis, DNA


  • Bacterial Proteins
  • CRISPR-Associated Protein 9