Abstract 16920: Permanent Reduction of Ryr2 Improves Lv Ejection Fraction in Heart Failure
Circulation(2018)
Abstract
Introduction: Ryanodine receptor type 2 (RyR-2), the main Ca 2+ release channel from sarcoplasmic reticulum in cardiomyocytes, plays an important role in regulation of myocardial contractile function and cardiac hypertrophy. Hypothesis: RyR-2 is a target of gene therapy in heart failure. Methods and Results: Two CRISPR-Cas9 gRNA targeting RyR-2 were identified: one located on the gene N terminal and the other on the C terminal. Adeno-associated virus was used to express CRISPR-Cas9 to knockdown RyR-2 in two weeks transverse aortic constriction (TAC) model of mice. After one week of administration of the virus, compared with the control, cardiac mass, heart size and fibrosis decreased and the LV ejection fraction increased significantly in the model with CRISPR-Cas9 gRNA on the RyR-2 N terminal, and the other one had a similar result but not significantly. Conclusions: Our results hint genome editing with CRISPR-Cas9 disrupts the RyR-2 in vivo with partly restore the function of the heart.
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