Recent advances in genome editing technology; engineered Cas proteins

Document Type : Original Article

Authors

1 Biological group, Farzanegan campus, Semnan university, Semnan, Iran

2 Department of Medical Genetics, School of Medicine, Iran University of Medical Sciences, Tehran, Iran

3 Biological group,, Farzanegan Campus, Semnan University, Semnan, Iran.

4 Department of Biology, Faculty of Basic Science, Ale Taha Institute of Higher Education, Tehran, Iran

Abstract
Clustered regularly interspaced short palindromic repeats (CRISPR) were discovered with the discovery of prokaryotic CRISPR-related proteins called Cas, which are capable of creating double-strand breaks in target sequences in the mammalian genome with the help of guide RNA. This discovery led to the development of CRISPR systems that harness natural DNA repair mechanisms to precisely create mutations in defective genes. CRISPR has been used to remove deleterious mutated genes and correct errors in protein-coding sequences to rescue disease phenotypes in preclinical studies and in several clinical trials. However, most genetic disorders result from a combination of other mutations involving deletions and duplications in both coding and non-coding regions of the genome and therefore require complex genome engineering strategies beyond simple gene deletion. To overcome this limitation, the toolbox of natural and engineered CRISPR-Cas systems has expanded dramatically to include a variety of tools. The use of CRISPR technology to edit non-coding genomes, regulate gene expression, make precise genetic changes, and target infectious diseases has the potential to lead to therapeutic solutions for many incurable diseases.

Keywords

Subjects


Articles in Press, Accepted Manuscript
Available Online from 25 August 2026

  • Receive Date 16 October 2025
  • Revise Date 05 August 2026
  • Accept Date 23 August 2026
  • Publish Date 25 August 2026