Applications and Advancements of CRISPR/Cas9 Technology: An Update.

Abstract

Objectives: The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system is an RNA-guided DNA targeting platform widely known for its application in genome editing. Originally derived from the bacterial and archaebacterial defense mechanism against phage infection, it has since been studied and utilized for its potential as a genetic engineering tool and as a therapeutic agent. The Cas9 protein in its standard form induces double-stranded breaks (DSBs) in the target dsDNA sequence; however, modifications of the Cas9 protein have allowed for single-stranded breaks (SSBs) and even epigenetic modifications of gene expression. In comparison with previous methods including RNA interference, Zinc Finger Nucleases, and TAL Effector Nucleases, CRISPR is cheaper, more easily customized, and has a higher fidelity to its target site with fewer off-target effects. Consequently, CRISPR has become a central gene editing technique in a broad variety of research settings, with great potential for applications in human health. In this review, we offer an overview of CRISPR's mechanism of action and recent advancements in the application of CRISPR, as well as discuss literature pertinent to CRISPR applications to human health including many exciting prospective treatments for serious pathologies.