Open AccessCharacterization of Recombinant Adeno-Associated Viral Transduction and Safety Profiles in Cardiomyocytes
Author(s) -
Ai Jianzhong,
He Yong,
Zheng Mingxia,
Wen Yi,
Zhang Huan,
Huang Fangyang,
Zhu Ye
Publication year - 2018
Publication title -
cellular physiology and biochemistry
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.486
H-Index - 87
eISSN - 1421-9778
pISSN - 1015-8987
DOI - 10.1159/000492510
Subject(s) - original paper
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.