The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Inherited Diseases

Sameh A Abdelnour; Long Xie; Abdallah A Hassanin; Erwei Zuo; Yangqing Lu

doi:10.3389/fcell.2021.699597

The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Inherited Diseases

Front Cell Dev Biol. 2021 Dec 15:9:699597. doi: 10.3389/fcell.2021.699597. eCollection 2021.

Authors

Sameh A Abdelnour^{1

2}, Long Xie³, Abdallah A Hassanin⁴, Erwei Zuo³, Yangqing Lu¹

Affiliations

¹ State Key Laboratory for Conservation and Utilization of Subtropical Agro-Bioresources, College of Animal Science and Technology, Guangxi University, Nanning, China.
² Animal Production Department, Faculty of Agriculture, Zagazig University, Zagazig, Egypt.
³ Center for Animal Genomics, Agricultural Genome Institute at Shenzhen, Chinese Academy of Agricultural Sciences, Shenzhen, China.
⁴ Genetics Department, Faculty of Agriculture, Zagazig University, Zagazig, Egypt.

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR) is a promising innovative technology for genomic editing that offers scientists the chance to edit DNA structures and change gene function. It has several possible uses consisting of editing inherited deficiencies, treating, and reducing the spread of disorders. Recently, reports have demonstrated the creation of synthetic RNA molecules and supplying them alongside Cas9 into genome of eukaryotes, since distinct specific regions of the genome can be manipulated and targeted. The therapeutic potential of CRISPR/Cas9 technology is great, especially in gene therapy, in which a patient-specific mutation is genetically edited, or in the treating of human disorders that are untreatable with traditional treatments. This review focused on numerous, in vivo, in vitro, and ex vivo uses of the CRISPR/Cas9 technology in human inherited diseases, discovering the capability of this versatile in medicine and examining some of the main limitations for its upcoming use in patients. In addition to introducing a brief impression of the biology of the CRISPR/Cas9 scheme and its mechanisms, we presented the utmost recent progress in the uses of CRISPR/Cas9 technology in editing and treating of human genetic diseases.

Keywords: CRISPR/Cas9; gene editing; genetic diseases; rectifying; treatment.

Publication types

Review