Advantages, Disadvantages and Risks of CRISPR/cas9 Technique for Gene Therapy

Radbakhsh, Shabnam; Namdari Moghaddam, Hoda

doi:10.22034/atj.2024.198823

Advantages, Disadvantages and Risks of CRISPR/cas9 Technique for Gene Therapy

Reviewers

Authors

Shabnam Radbakhsh ¹

Hoda Namdari Moghaddam ²

¹ Department of Medical Biotechnology, School of Medicine Mashhad University of Medical Sciences, Mashhad, Iran

² Department of Virology, School of Public Health, Tehran University of Medical Sciences, Tehran, 1417613151, Iran

10.22034/atj.2024.198823

Abstract

The CRISPR/Cas9 system has gained significant attention as a gene editing method in recent years because of its simple design, cost-effectiveness, high efficiency, and ease of use. Additionally, it allows for the simultaneous editing of many locations. Additionally, it may be performed without the use of plasmids, so avoiding the many complications associated with plasmids. CRISPR/Cas9 has shown significant promise in the investigation of genes and genomic activities in microbes, plants, animals, and humans. Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are genetic disorders characterized by severe and possibly life-threatening symptoms. In this article, we discuss the use of CRISPR/Cas9 technology in the treatment of these two diseases and FDA-approved drugs based on CRISPR/Cas9. In addition, we address the most important challenges of gene therapy using this technology.

Keywords

CRISPR/Cas9

Gene therapy

β-thalassemia

Sickle cell disease