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

Author:

Zhou Jun12,Ren Zhuoying12,Xu Jie1,Zhang Jifeng1,Chen Y. Eugene12ORCID

Affiliation:

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

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).

Publisher

Walter de Gruyter GmbH

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