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Cardiac Delivery of Nucleic Acids by Transcriptional and Transductional Targeting of Adeno-Associated Viral Vectors

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RNA Technologies in Cardiovascular Medicine and Research

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Abstract

RNAi-based approaches show promising effects in preclinical models of cardiac diseases. However, a potential clinical use may be limited by the low efficiency of cardiac transfer. Efficient and sustained cardiac delivery in large animal models and finally clinical trials requires transfer of vectorized shRNA with suitable application systems. Packaging shRNA constructs in targeted adeno-associated viral (AAV) vectors may be versatile for systemic delivery into rodent hearts. Expressing microRNA under control of cardiac-specific promoters may further increase efficiency and specificity of delivery. For large animals and finally clinical studies, careful selection of the vector and application system will be necessary to obtain valid results.

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  1. Innere Medizin III, Universitatsklinikum Heidelberg, Im Neuenheimer Feld 410, 69120, Heidelberg, Germany

    O. J. Müller & H. A. Katus

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  1. O. J. Müller
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  2. H. A. Katus
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  1. Institute for Chemistry/Biochemistry, Free University of Berlin, Thielallee 63, 14195, Berlin, Germany

    Volker A. Erdmann

  2. Clinic for Cardiology and Pneumology Campus Benjamin Franklin, Charite University Medicine Berlin, Hindenburgdamm 30, 12200, Berlin, Germany

    Wolfgang Poller

  3. Institute of Bioorganic Chemistry of the Polish Academy of Sciences, Noskowskiego 12, 61-704, Poznan, Poland

    Jan Barciszewski

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Müller, O.J., Katus, H.A. (2008). Cardiac Delivery of Nucleic Acids by Transcriptional and Transductional Targeting of Adeno-Associated Viral Vectors. In: Erdmann, V.A., Poller, W., Barciszewski, J. (eds) RNA Technologies in Cardiovascular Medicine and Research. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-540-78709-9_9

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