title: AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.
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.
authors.affiliation: Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina 27599; email: rjs@med.unc.edu.
authors.forename: R Jude
authors.initials: RJ
authors.lastname: Samulski
authors.affiliation: Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida 32610; email: nmuzyczk@ufl.edu.
authors.forename: Nicholas
authors.initials: N
authors.lastname: Muzyczka
keywords: DNA transfer; gene therapy; parvovirus; transduction; viral vector