articleAnnual Review of VirologyOct 1, 2014BRONZE OA

AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

RJR. Jude SamulskiNMNicholas Muzyczka

University of North Carolina at Chapel Hill · University of Florida · +1 more institution

PubMed
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Abstract

Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species and tissues in vivo with no evidence of toxicity, and it generates relatively mild innate and adaptive immune responses. We review the basic biology of AAV, the history of progress in AAV vector technology, and some of the clinical and research applications where AAV has shown success.

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Topics & keywords

Topics
Keywords
  • Biology
  • Adeno-associated virus
  • Genetic enhancement
  • Gene transfer
  • Vector (molecular biology)
  • Virus
  • Virology
  • Innate immune system
UN Sustainable Development Goals
  • Life in Land
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