Characterization of Recombinant Adeno-Associated Viral Transduction and Safety Profiles in Cardiomyocytes
Characterization of Recombinant Adeno-Associated Viral Transduction and Safety Profiles in Cardiomyocytes
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DOI：
10.1159/000492510
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发表时间：
2018-01-01
影响因子：
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通讯作者：
Zhu, Ye
中科院分区：
文献类型：
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作者：
Ai, Jianzhong;He, Yong;Zhu, Ye
背景/目的：心血管疾病（CVD）是人类死亡的主要原因。然而，对这些疾病的有效治疗仍然缺乏。目前，基因治疗可能是一种有效治疗心脏病的潜在方法。本研究的目的是分析重组腺相关病毒（AAV）血清9型在体内和体外对心肌细胞的转导效率和安全性。研究方法：我们生产的rAAV血清型9表达增强型绿色荧光蛋白（EGFP）驱动的心肌肌钙蛋白T（cTNT）启动子，并表征其在体外原代培养的心肌细胞，在野生型小鼠心脏组织在体内的转导效率。结果：rAAV 9在体外可有效转导小鼠心肌细胞。静脉注射后，rAAV 9可以有效和安全地转染与心脏疾病有关的心肌细胞。结论：rAAV 9能安全、有效地在体外和/或体内转染心肌细胞。rAAV 9血清型载体可能构成未来心脏病基因治疗的强大工具箱。(C)2018作者（S）由S发布。Karger AG，巴塞尔
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