Characterization of Recombinant Adeno-Associated Viral Transduction and Safety Profiles in Cardiomyocytes

Jianzhong Ai, Yong He, Mingxia Zheng, Yi Wen, Huan Zhang, Fangyang Huang, Ye Zhu
2018 Cellular Physiology and Biochemistry  
Background/Aims: Cardiovascular diseases (CVD) are the leading causes for human mortality. However, the effective treatment for these diseases are still lacking. Currently, gene therapy could be a potential way for efficiently treating heart diseases. The aim of our study is to analyze the transduction efficacy and safety profile of recombinant adeno associated virus (AAV) serotype 9 for cardiomyocytes in vivo and in vitro. Methods: We produced rAAV serotype 9 expressing enhanced green
more » ... nce protein (EGFP) driven by a cardiac troponin T (cTNT) promoter, and characterized its transduction efficiency in primary cultured cardiomyocytes in vitro, and in wild-type mouse heart tissue in vivo. Results: Our data showed that rAAV9 efficiently transduced mouse cardiomyocytes in vitro. Following intravenous injection, rAAV9 could efficiently and safely transduce cardiomyocytes that are involved in heart diseases. Conclusion: Our findings suggested that rAAV9 can efficiently and safely transduce cardiomyocytes in vitro and/or in vivo. The rAAV9 serotype vector could constitute a powerful toolbox for future gene therapy of heart diseases. Fig. 7. rAAV9-cTNT efficiently transduce cardiomyocytes in vitro. The majority of cells that showing green fluorescence were positively stained with cTNT. The white arrowhead showed the cells with positive EGFP fluorescence and cTNT staining.
doi:10.1159/000492510 pmid:30092576 fatcat:lncrfslqunctjbtjbnq6pxiade