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Curr Opin Virol. 2016 Dec;21:54-60. doi: 10.1016/j.coviro.2016.08.001. Epub 2016 Aug 18.

Cellular transduction mechanisms of adeno-associated viral vectors.

Author information

1
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Department of Genetics, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Curriculum in Genetics and Molecular Biology, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
2
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Department of Genetics, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Department of Biochemistry & Biophysics, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. Electronic address: aravind@med.unc.edu.

Abstract

Recombinant adeno-associated viral vectors (rAAV) are regarded as promising vehicles for therapeutic gene delivery. Continued development and new strategies are essential to improve the potency of AAV vectors and reduce the effective dose needed for clinical efficacy. In this regard, many studies have focused on understanding the cellular transduction mechanisms of rAAV, often with the goal of exploiting this knowledge to increase gene transfer efficiency. Here, we provide an overview of our evolving understanding of rAAV cellular trafficking pathways through the host cell, beginning with cellular entry and ending with transcription of the vector genome. Strategies to exploit this information for improving rAAV transduction are discussed.

PMID:
27544821
PMCID:
PMC5138113
DOI:
10.1016/j.coviro.2016.08.001
[Indexed for MEDLINE]
Free PMC Article

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