MicroRNA-122–Mediated Liver Detargeting Enhances the Tissue Specificity of Cardiac Genome Editing

作者: Luzi Yang, Gonglie Chen, Congting Guo, Qinghua Cui, Xiaomin Hu, Yifei Li, Liang Chen, William T. Pu and Yuxuan Guo
杂志: Circulation
出版日期: 2024 May 28

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摘要

Recombinant adeno-associated virus(rAAV) is broadly applied in cardiovascular research and gene therapy: Although AAV serotype 9 (AAV9) robustly transduces the liver, the heart, and other organs, its gene expression can be targeted to cardiomyocytes using a specific promoter usually from the cardiac troponin T gene (Tnnt2/cTnT). This AAv9-Tnnt2 system is heavily used in research, but its cardiac specificity relative to other organs requires greater investigation. This issue is especially important for applications where low or transient gene expression is sufficient to cause profound biological outcomes.

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