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

被引：6
作者：
Ai, Jianzhong [1 ]
He, Yong [2 ]
Zheng, Mingxia [2 ]
Wen, Yi [2 ]
Zhang, Huan [2 ]
Huang, Fangyang [2 ]
Zhu, Ye [2 ]
机构：
[1] Sichuan Univ, West China Hosp, Dept Urol, Inst Urol, Chengdu, Sichuan, Peoples R China
[2] Sichuan Univ, West China Hosp, Dept Cardiol, Guoxue Xiang 37, Chengdu 610041, Sichuan, Peoples R China
来源：
CELLULAR PHYSIOLOGY AND BIOCHEMISTRY | 2018年 / 48卷 / 05期
基金：
中国国家自然科学基金; 国家重点研发计划;
关键词：
Adeno-associated virus; Safety profile; Gene therapy; Heart diseases; Transduction efficacy; GENE-THERAPY; RENEWAL;
D O I：
10.1159/000492510
中图分类号：
Q2 [细胞生物学];
学科分类号：
071009 ; 090102 ;
摘要：
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 fluorescence 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. (C) 2018 The Author(s) Published by S. Karger AG, Basel
引用
收藏
页码：1894 / 1900
页数：7
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