Therapeutic Genome Editing by CRISPR/Cas9-Mediated Strategy to Cure Genetic Disorders in Humans: Guide for Molecular Surgeons

Crit Rev Eukaryot Gene Expr. 2019;29(5):387-399. doi: 10.1615/CritRevEukaryotGeneExpr.2019025930.

Abstract

Genetic engineering scientists have shown a wide increase especially on therapeutic promise which has gained considerable awareness. This goes back to a variety of tools including the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9) system. This technique has shown an efficient flourish in genome editing and treatment of genetic diseases. Due to its specificity and ease of use it has been widely adopted for a variety of applications involving human diseases, treatment, therapeutics in cancer and biotechnology. Albeit studies have shown that CRISPR/Cas9 technique is more proficient, particular and manipulable than past eras of genetic modulation instruments. Additionally, it can be further enhanced by the application of different developments raising its general productivity in a higher recurrence of genome modifications and decreasing its off-target impacts. Here in this review this technique will be introduced with discussing its relation with the genome engineering and gene editing especially in the therapeutic area. Furthermore, the specificity of the application is explained and the contribution of Induced Human Pluripotent Stem Cells (iPSCs) in gene manipulation and how CRISPR technology expanded its application is discussed. Lastly, we describe the novel effective applications of the CRISPR/Cas9 technology and propose some enhancements that might improve the technique with highlighting the future approach and remarks.

Publication types

  • Review

MeSH terms

  • CRISPR-Cas Systems*
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics
  • Gene Editing*
  • Genetic Diseases, Inborn / genetics
  • Genetic Diseases, Inborn / therapy*
  • Genetic Engineering
  • Genetic Therapy*
  • Humans
  • Induced Pluripotent Stem Cells
  • Surgeons