Gene-Edited Human Pluripotent Stem Cells for Cardiac Disease Research

MA Shuhong^1,2,3, WANG Yongming⁴*, LAN Feng^1,2,3*

(¹State Key Laboratory of Cardiovascular Disease, National Center for Cardiovascular Diseases, Fuwai Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 102308, China; ²Beijing Laboratory for Cardiovascular Precision Medicine, the Key Laboratory of Biomedical Engineering for Cardiovascular Disease Research, the Key Laboratory of Remodeling-Related Cardiovascular Disease, Ministry of Education, Beijing Anzhen Hospital, Capital Medical University, Beijing 100029, China; ³Beijing Institute of Heart, Lung and Blood Vessel Diseases, Beijing 100029, China; ⁴State Key Laboratory of Genetic Engineering, School of Life Sciences, Fudan University, Shanghai 200432, China)

Abstract:

CVD (cardiovascular disease) is one of the leading causes of human death. In vitro cell model can improve the ability to study these diseases. The multi-differentiation ability of hPSC (human pluripotent stem cell) provides the possibility to establish various cell or tissue models. Recently, the development of several novel genome editing tools, including the CRISPR/Cas system, BE (base editor) and PE (prime editor) have greatly improved the efficiency of manufacturing and testing cell disease models. The combination of human pluripotent stem cell and genome editing technology provides new startegies for exploring and curing diseases. This review focuses on the pro and cons of the most popular genome editing technologies, as well as their potential applications in cardiovascular disease.

CSTR: 32200.14.cjcb.2021.07.0006