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Hum Gene Ther. 2013 Nov;24(11):906-13. doi: 10.1089/hum.2013.2515.

An emerging adeno-associated viral vector pipeline for cardiac gene therapy.

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1 Gene Therapy Center, The University of North Carolina at Chapel Hill , Chapel Hill, NC 27516.


The naturally occurring adeno-associated virus (AAV) isolates display diverse tissue tropisms in different hosts. Robust cardiac transduction in particular has been reported for certain AAV strains. Successful applications of these AAV strains in preclinical and clinical settings with a focus on treating cardiovascular disease continue to be reported. At the same time, these studies have highlighted challenges such as cross-species variability in AAV tropism, transduction efficiency, and immunity. Continued progress in our understanding of AAV capsid structure and biology has provided the rationale for designing improved vectors that can possibly address these concerns. The current report provides an overview of cardiotropic AAV, existing gaps in our knowledge, and newly engineered AAV strains that are viable candidates for the cardiac gene therapy clinic.

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