Improvements of nuclease and nickase gene modification techniques for the treatment of genetic diseases

Yaoyao Lu; Cedric Happi Mbakam; Bo Song; Eli Bendavid; Jacques-P Tremblay

doi:10.3389/fgeed.2022.892769

Improvements of nuclease and nickase gene modification techniques for the treatment of genetic diseases

Front Genome Ed. 2022 Jul 26:4:892769. doi: 10.3389/fgeed.2022.892769. eCollection 2022.

Authors

Yaoyao Lu^{1

2}, Cedric Happi Mbakam^{1

2}, Bo Song^{1

2}, Eli Bendavid^{1

2}, Jacques-P Tremblay^{1

2}

Affiliations

¹ CHU de Québec Research Center, Laval University, Quebec City, QC, Canada.
² Department of Molecular Medicine, Laval University, Quebec City, QC, Canada.

Abstract

Advancements in genome editing make possible to exploit the functions of enzymes for efficient DNA modifications with tremendous potential to treat human genetic diseases. Several nuclease genome editing strategies including Meganucleases (MNs), Zinc Finger Nucleases (ZFNs), Transcription Activator-like Effector Nucleases (TALENs) and Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR associated proteins (CRISPR-Cas) have been developed for the correction of genetic mutations. CRISPR-Cas has further been engineered to create nickase genome editing tools including Base editors and Prime editors with much precision and efficacy. In this review, we summarized recent improvements in nuclease and nickase genome editing approaches for the treatment of genetic diseases. We also highlighted some limitations for the translation of these approaches into clinical applications.

Keywords: Adenosine Base Editor (ABE); CRISPR-cas; Cytidine Base Editor (CBE); TALEN; ZFN; gene editing; prime editing.

Publication types

Review