Nuclease-Deficient Clustered Regularly Interspaced Short Palindromic Repeat-Based Approaches for In Vitro and In Vivo Gene Activation

Angela Lek; Kaiyue Ma; Keryn G Woodman; Monkol Lek

doi:10.1089/hum.2020.241

Nuclease-Deficient Clustered Regularly Interspaced Short Palindromic Repeat-Based Approaches for In Vitro and In Vivo Gene Activation

Hum Gene Ther. 2021 Mar;32(5-6):260-274. doi: 10.1089/hum.2020.241.

Authors

Angela Lek¹, Kaiyue Ma¹, Keryn G Woodman¹, Monkol Lek¹

Affiliation

¹ Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA.

PMID: 33446040
DOI: 10.1089/hum.2020.241

Abstract

Clustered regularly interspaced short palindromic repeat (CRISPR)-based technology has been adapted to achieve a wide range of genome modifications, including transcription regulation. The focus of this review is on the application of CRISPR-based platforms such as nuclease-deficient Cas9 and Cas12a, to achieve targeted gene activation. We review studies to date that have used CRISPR-based activation technology for the elucidation of biological mechanism and disease correction, as well as its application in genetic screens as a powerful tool for high-throughput genotype-phenotype mapping. In addition to our synthesis and critical analysis of published studies, we explore key considerations for the potential clinical translation of CRISPR-based activation technology.

Keywords: CRISPR; gene activation; gene upregulation.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

CRISPR-Cas Systems* / genetics
Clustered Regularly Interspaced Short Palindromic Repeats / genetics
Endonucleases
Gene Editing*
Transcriptional Activation

Substances

Endonucleases