-Review-

Serotype-independent Method of Recombinant Adeno-associated Virus (AAV) Vector Production and Purification

Koichi Miyake, Noriko Miyake, Yoshiyuki Yamazaki, Takashi Shimada and Yukihiko Hirai

Department of Biochemistry and Molecular Biology, Division of Gene Therapy Research Center for Advanced Medical Technology, Nippon Medical School

A variety of gene transfer strategies have been developed to treat inherited, degenerative, and acquired diseases. Among the different vector systems developed so far, recombinant adeno-associated viral (AAV) vectors have shown notable benefits, including prolonged gene expression, transduction of both dividing and nondividing cells, and a lack of pathogenicity caused by wild-type infections. Thanks to these features, the use of AAV vectors as a gene transfer tool has increased dramatically during the past several years, and several recent clinical trials have used AAV vectors. However, AAV vectors are more complicated to produce than are other viral vectors. With steady advances toward clinical application, much effort has been made to isolate novel AAV serotypes and to develop methods for their efficient, scalable, and versatile production and purification. Here we review state of the art methods for AAV vector production and purification, which we have refined in our laboratory.

J Nippon Med Sch 2012; 79: 394-402

Keywords
AAV vector, Serotype, Self-complementary AAV, Purification

Correspondence to
Koichi Miyake, MD, PhD, Department of Biochemistry and Molecular Biology, Nippon Medical School, 1-1-5 Sendagi, Bunkyo-ku, Tokyo 113-8602, Japan
kmiyake@nms.ac.jp

Received, October 26, 2012
Accepted, November 27, 2012