AAV-Mediated Gene Therapy for Research and Therapeutic Purposes R. Jude Samulski 1 and Nicholas Muzyczka 2 1 Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina 27599; email: rjs@med.unc.edu 2 Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida 32610; email: nmuzyczk@ufl.edu Annu. Rev. Virol. 2014. 1:427–51 The Annual Review of Virology is online at virology.annualreviews.org This article’s doi: 10.1146/annurev-virology-031413-085355 Copyright c  2014 by Annual Reviews. All rights reserved Keywords viral vector, DNA transfer, parvovirus, gene therapy, transduction 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. 427 Annual Review of Virology 2014.1:427-451. Downloaded from www.annualreviews.org by 98.156.86.215 on 10/05/14. For personal use only.