Gene editing therapy ready for cardiovascular diseases: opportunities, challenges, and perspectives

Jun Zhou; Zhuoying Ren; Jie Xu; Jifeng Zhang; Y Eugene Chen

doi:10.1515/mr-2021-0010

Gene editing therapy ready for cardiovascular diseases: opportunities, challenges, and perspectives

Med Rev (2021). 2021 Oct 21;1(1):6-9. doi: 10.1515/mr-2021-0010. eCollection 2021 Oct.

Authors

Jun Zhou^{1

2}, Zhuoying Ren^{1

2}, Jie Xu¹, Jifeng Zhang¹, Y Eugene Chen^{1

2}

Affiliations

¹ Center for Advanced Models for Translational Sciences and Therapeutics, University of Michigan Medical Center, Ann Arbor 48109, MI, USA.
² Department of Pharmacology, University of Michigan Medical Center, Ann Arbor 48109, MI, USA.

Abstract

Gene editing nucleases (GENs), represented by CRISPR/Cas9, have become major tools in biomedical research and offer potential cures for many human diseases. Gene editing therapy (GETx) studies in animal models targeting genes such as proprotein convertase subtilisin/kexin type 9 (PCSK9), apolipoprotein C3 (APOC3), angiopoietin Like 3 (ANGPTL3) and inducible degrader of the low-density lipoprotein receptor (IDOL) have demonstrated the benefits and advantages of GETx in managing atherosclerosis. Here we present our views on this brand new therapeutic option for cardiovascular diseases (CVD).

Keywords: CRISPR/Cas9; cardiovascular diseases; gene editing therapy.